Donald Barry Kohn

Title(s)Professor, MIMG
SchoolMedicine
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    Other Positions
    Title(s)Professor, Molecular and Medical Pharmacology

    Title(s)Professor, Pediatrics


    Collapse Research 
    Collapse Research Activities and Funding
    Disseminating Curative Biological Therapies for Rare Pediatric Diseases
    NIH U01TR001814Sep 16, 2016 - Jun 30, 2021
    Role: Co-Principal Investigator
    A Phase II/III Clinical Trial of Gene Therapy for ADA-Deficient SCID
    NIH/NIAID U01AI122260Mar 1, 2016 - Feb 28, 2022
    Role: Principal Investigator
    EFS-ADA Lentiviral Vector Transduction of Bone Marrow CD34+ Cells for ADA-SCID
    NIH U01AI100801Aug 1, 2012 - Jul 31, 2018
    Role: Principal Investigator
    Primary Immune Deficiency Treatment Consortium
    NIH U54AI082973Sep 12, 2009 - Aug 31, 2024
    Role: Co-Principal Investigator
    In Vivo ADA Gene Delivery for the Treatment of SCID
    NIH R01AI074043Jun 1, 2007 - May 31, 2013
    Role: Principal Investigator
    Training in Developmental Hematology
    NIH T32HL086345Apr 1, 2007 - May 31, 2023
    Role: Principal Investigator
    Clinical Trial Planning Grant: Lentiviral Vector for HIV-1
    NIH R34AI069947May 15, 2006 - Oct 30, 2007
    Role: Principal Investigator
    MND-ADA Transduced CD34 Cells for ADA-SCID
    NIH R01FD003005Sep 30, 2005 - Feb 28, 2013
    Role: Principal Investigator
    Conference--American Society of Gene Therapy
    NIH R13HL078311Jul 1, 2004 - Jun 30, 2009
    Role: Principal Investigator
    Gene Therapy Using Hematopoietic Stem Cells
    NIH P01HL073104Apr 1, 2003 - Jun 30, 2015
    Role: Principal Investigator
    GENE EXPRESSION IN BETA-CELLS BY LENTIVIRAL VECTORS
    NIH R21DK062649Jul 1, 2002 - Jun 30, 2004
    Role: Principal Investigator
    LENTIVIRAL VECTOR TRANSFER TO HEMATOPOIETIC STEM CELLS
    NIH R01AI052798Jun 1, 2002 - May 31, 2007
    Role: Principal Investigator
    MODIFIED RETROVIRAL VECTORS FOR HSC GENE THERAPY
    NIH R01DK056287Aug 1, 1999 - Jul 31, 2003
    Role: Principal Investigator
    GENE THERAPY FOR HURLERS DISEASE
    NIH R01DK054566Sep 5, 1998 - May 31, 2002
    Role: Principal Investigator
    IN VIVO MODEL OF HUMAN STEM CELL GENE THERAPY
    NIH R01DK048700Sep 30, 1994 - Aug 31, 1998
    Role: Principal Investigator
    MODIFIED RETROVIRAL VECTORS FOR STABLE GENE EXPRESSION
    NIH R01DK049000Sep 30, 1994 - Aug 31, 1997
    Role: Principal Investigator
    GLUCOCEREBROSIDASE GENE EXPRESSION--HEMATOPOIETIC CELLS
    NIH R01DK042694Sep 1, 1991 - Aug 31, 1996
    Role: Principal Investigator
    RETROVIRAL VECTOR-MEDIATED TRANSFER OF NEF GENE
    NIH R01AI029125Sep 30, 1989 - Jul 31, 1993
    Role: Principal Investigator

    Collapse ORNG Applications 
    Collapse Clinical Trials

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy. J Clin Immunol. 2022 Apr 21. Arnold DE, Chellapandian D, Parikh S, Mallhi K, Marsh RA, Heimall JR, Grossman D, Chitty-Lopez M, Murguia-Favela L, Gennery AR, Boulad F, Arbuckle E, Cowan MJ, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson T, Kang EM, Malech HL, Leiding JW. PMID: 35445907; PMCID: PMC9022412.
      View in: PubMed   Mentions:
    2. Previous Infection Combined with Vaccination Produces Neutralizing Antibodies with Potency against SARS-CoV-2 Variants. mBio. 2021 12 21; 12(6):e0265621. Ibarrondo FJ, Hofmann C, Ali A, Ayoub P, Kohn DB, Yang OO. PMID: 34872349; PMCID: PMC8649753.
      View in: PubMed   Mentions:
    3. Improved lentiviral vector titers from a multi-gene knockout packaging line. Mol Ther Oncolytics. 2021 Dec 17; 23:582-592. Han J, Tam K, Tam C, Hollis RP, Kohn DB. PMID: 34938858; PMCID: PMC8660686.
      View in: PubMed   Mentions:
    4. Development of allogeneic HSC-engineered iNKT cells for off-the-shelf cancer immunotherapy. Cell Rep Med. 2021 11 16; 2(11):100449. Li YR, Zhou Y, Kim YJ, Zhu Y, Ma F, Yu J, Wang YC, Chen X, Li Z, Zeng S, Wang X, Lee D, Ku J, Tsao T, Hardoy C, Huang J, Cheng D, Montel-Hagen A, Seet CS, Crooks GM, Larson SM, Sasine JP, Wang X, Pellegrini M, Ribas A, Kohn DB, Witte O, Wang P, Yang L. PMID: 34841295; PMCID: PMC8607011.
      View in: PubMed   Mentions:
    5. Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency. Blood. 2021 10 14; 138(15):1304-1316. Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. PMID: 33974038; PMCID: PMC8525336.
      View in: PubMed   Mentions: 2     Fields:    
    6. Regional Gene Therapy with Transduced Human Cells: The Influence of "Cell Dose" on Bone Repair. Tissue Eng Part A. 2021 11; 27(21-22):1422-1433. Ihn H, Kang H, Iglesias B, Sugiyama O, Tang A, Hollis R, Bougioukli S, Skorka T, Park S, Longjohn D, Oakes DA, Kohn DB, Lieberman JR. PMID: 33882718; PMCID: PMC8827114.
      View in: PubMed   Mentions:    Fields:    
    7. Evidence generation and reproducibility in cell and gene therapy research: A call to action. Mol Ther Methods Clin Dev. 2021 Sep 10; 22:11-14. Abou-El-Enein M, Angelis A, Appelbaum FR, Andrews NC, Bates SE, Bierman AS, Brenner MK, Cavazzana M, Caligiuri MA, Clevers H, Cooke E, Daley GQ, Dzau VJ, Ellis LM, Fineberg HV, Goldstein LSB, Gottschalk S, Hamburg MA, Ingber DE, Kohn DB, Krainer AR, Maus MV, Marks P, Mummery CL, Pettigrew RI, Rutter JL, Teichmann SA, Terzic A, Urnov FD, Williams DA, Wolchok JD, Lawler M, Turtle CJ, Bauer G, Ioannidis JPA. PMID: 34377737; PMCID: PMC8322039.
      View in: PubMed   Mentions:
    8. Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy. J Clin Immunol. 2021 10; 41(7):1597-1606. Baloh CH, Borkar SA, Chang KF, Yao J, Hershfield MS, Parikh SH, Kohn DB, Goodenow MM, Sleasman JW, Yin L. PMID: 34184208.
      View in: PubMed   Mentions:    Fields:    
    9. Regional gene therapy for bone healing using a 3D printed scaffold in a rat femoral defect model. J Biomed Mater Res A. 2021 11; 109(11):2346-2356. Kang HP, Ihn H, Robertson DM, Chen X, Sugiyama O, Tang A, Hollis R, Skorka T, Longjohn D, Oakes D, Shah R, Kohn D, Jakus AE, Lieberman JR. PMID: 34018305.
      View in: PubMed   Mentions:    Fields:    
    10. Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. N Engl J Med. 2021 05 27; 384(21):2002-2013. Kohn DB, Booth C, Shaw KL, Xu-Bayford J, Garabedian E, Trevisan V, Carbonaro-Sarracino DA, Soni K, Terrazas D, Snell K, Ikeda A, Leon-Rico D, Moore TB, Buckland KF, Shah AJ, Gilmour KC, De Oliveira S, Rivat C, Crooks GM, Izotova N, Tse J, Adams S, Shupien S, Ricketts H, Davila A, Uzowuru C, Icreverzi A, Barman P, Campo Fernandez B, Hollis RP, Coronel M, Yu A, Chun KM, Casas CE, Zhang R, Arduini S, Lynn F, Kudari M, Spezzi A, Zahn M, Heimke R, Labik I, Parrott R, Buckley RH, Reeves L, Cornetta K, Sokolic R, Hershfield M, Schmidt M, Candotti F, Malech HL, Thrasher AJ, Gaspar HB. PMID: 33974366; PMCID: PMC8240285.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCellsCTClinical Trials
    11. Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia. CRISPR J. 2021 04; 4(2):191-206. Gray DH, Villegas I, Long J, Santos J, Keir A, Abele A, Kuo CY, Kohn DB. PMID: 33876953; PMCID: PMC8336228.
      View in: PubMed   Mentions:    Fields:    
    12. Antiviral drug screen identifies DNA-damage response inhibitor as potent blocker of SARS-CoV-2 replication. Cell Rep. 2021 04 06; 35(1):108940. Garcia G, Sharma A, Ramaiah A, Sen C, Purkayastha A, Kohn DB, Parcells MS, Beck S, Kim H, Bakowski MA, Kirkpatrick MG, Riva L, Wolff KC, Han B, Yuen C, Ulmert D, Purbey PK, Scumpia P, Beutler N, Rogers TF, Chatterjee AK, Gabriel G, Bartenschlager R, Gomperts B, Svendsen CN, Betz UAK, Damoiseaux RD, Arumugaswami V. PMID: 33784499; PMCID: PMC7969873.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    13. Gene Therapies for Primary Immune Deficiencies. Front Immunol. 2021; 12:648951. Kohn LA, Kohn DB. PMID: 33717203; PMCID: PMC7946985.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    14. Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID. Mol Ther Methods Clin Dev. 2021 Mar 12; 20:765-778. Carbonaro-Sarracino DA, Chun K, Clark DN, Kaufman ML, Jin X, Wang X, Kohn DB. PMID: 33738330; PMCID: PMC7940710.
      View in: PubMed   Mentions: 1  
    15. Correction to: Infections in Infants with SCID: Isolation, Infection Screening and Prophylaxis in PIDTC Centers. J Clin Immunol. 2021 Feb; 41(2):498-500. Dorsey M, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Burroughs LM, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier G, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J. PMID: 33274413.
      View in: PubMed   Mentions:    Fields:    
    16. Safe and Effective In Vivo Targeting and Gene Editing in Hematopoietic Stem Cells: Strategies for Accelerating Development. Hum Gene Ther. 2021 01; 32(1-2):31-42. Cannon P, Asokan A, Czechowicz A, Hammond P, Kohn DB, Lieber A, Malik P, Marks P, Porteus M, Verhoeyen E, Weissman D, Weissman I, Kiem HP. PMID: 33427035; PMCID: PMC8917879.
      View in: PubMed   Mentions: 1     Fields:    
    17. Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells. Front Genome Ed. 2020; 2:601541. Benitez EK, Lomova Kaufman A, Cervantes L, Clark DN, Ayoub PG, Senadheera S, Osborne K, Sanchez JM, Crisostomo RV, Wang X, Reuven N, Shaul Y, Hollis RP, Romero Z, Kohn DB. PMID: 34713224; PMCID: PMC8525354.
      View in: PubMed   Mentions:
    18. β-Globin Lentiviral Vectors Have Reduced Titers due to Incomplete Vector RNA Genomes and Lowered Virion Production. Stem Cell Reports. 2021 01 12; 16(1):198-211. Han J, Tam K, Ma F, Tam C, Aleshe B, Wang X, Quintos JP, Morselli M, Pellegrini M, Hollis RP, Kohn DB. PMID: 33186538; PMCID: PMC7897704.
      View in: PubMed   Mentions:
    19. Correction: Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2020 Nov; 40(8):1211-1213. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Martinez C, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium. PMID: 32860171.
      View in: PubMed   Mentions:    Fields:    
    20. Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies. Sci Adv. 2020 Oct; 6(43). Yang P, Chou SJ, Li J, Hui W, Liu W, Sun N, Zhang RY, Zhu Y, Tsai ML, Lai HI, Smalley M, Zhang X, Chen J, Romero Z, Liu D, Ke Z, Zou C, Lee CF, Jonas SJ, Ban Q, Weiss PS, Kohn DB, Chen K, Chiou SH, Tseng HR. PMID: 33097539; PMCID: PMC7608838.
      View in: PubMed   Mentions: 3     Fields:    
    21. Infections in Infants with SCID: Isolation, Infection Screening, and Prophylaxis in PIDTC Centers. J Clin Immunol. 2021 01; 41(1):38-50. Dorsey MJ, Wright NAM, Chaimowitz NS, Dávila Saldaña BJ, Miller H, Keller MD, Thakar MS, Shah AJ, Abu-Arja R, Andolina J, Aquino V, Barnum JL, Bednarski JJ, Bhatia M, Bonilla FA, Butte MJ, Bunin NJ, Chandra S, Chaudhury S, Chen K, Chong H, Cuvelier GDE, Dalal J, DeFelice ML, DeSantes KB, Forbes LR, Gillio A, Goldman F, Joshi AY, Kapoor N, Knutsen AP, Kobrynski L, Lieberman JA, Leiding JW, Oshrine B, Patel KP, Prockop S, Quigg TC, Quinones R, Schultz KR, Seroogy C, Shyr D, Siegel S, Smith AR, Torgerson TR, Vander Lugt MT, Yu LC, Cowan MJ, Buckley RH, Dvorak CC, Griffith LM, Haddad E, Kohn DB, Logan B, Notarangelo LD, Pai SY, Puck J, Pulsipher MA, Heimall J. PMID: 33006109; PMCID: PMC8388237.
      View in: PubMed   Mentions: 1     Fields:    
    22. Adenosine Deaminase (ADA)-Deficient Severe Combined Immune Deficiency (SCID) in the US Immunodeficiency Network (USIDNet) Registry. J Clin Immunol. 2020 11; 40(8):1124-1131. Kuo CY, Garabedian E, Puck J, Cowan MJ, Sullivan KE, Buckley RH, Cunningham-Rundles C, Marsh R, Candotti F, Kohn DB. PMID: 32880085; PMCID: PMC8216639.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansPHPublic Health
    23. Overview of the current status of gene therapy for primary immune deficiencies (PIDs). J Allergy Clin Immunol. 2020 08; 146(2):229-233. Kuo CY, Kohn DB. PMID: 32771134.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansAnimals
    24. AT1R Activating Autoantibodies in Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2020 11; 26(11):2061-2067. Bradford KL, Pearl M, Kohn DB, Weng P, Yadin O, Bowles V, De Oliveira SN, Moore TB. PMID: 32736008; PMCID: PMC8128374.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    25. Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy. Biol Blood Marrow Transplant. 2020 10; 26(10):1819-1827. Bradford KL, Liu S, Krajinovic M, Ansari M, Garabedian E, Tse J, Wang X, Shaw KL, Gaspar HB, Candotti F, Kohn DB. PMID: 32653625; PMCID: PMC7529956.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    26. Human CLEC9A antibodies deliver NY-ESO-1 antigen to CD141+ dendritic cells to activate naïve and memory NY-ESO-1-specific CD8+ T cells. J Immunother Cancer. 2020 07; 8(2). Masterman KA, Haigh OL, Tullett KM, Leal-Rojas IM, Walpole C, Pearson FE, Cebon J, Schmidt C, O'Brien L, Rosendahl N, Daraj G, Caminschi I, Gschweng EH, Hollis RP, Kohn DB, Lahoud MH, Radford KJ. PMID: 32737142; PMCID: PMC7394304.
      View in: PubMed   Mentions:
    27. Artificial thymic organoids represent a reliable tool to study T-cell differentiation in patients with severe T-cell lymphopenia. Blood Adv. 2020 06 23; 4(12):2611-2616. Bosticardo M, Pala F, Calzoni E, Delmonte OM, Dobbs K, Gardner CL, Sacchetti N, Kawai T, Garabedian EK, Draper D, Bergerson JRE, DeRavin SS, Freeman AF, Güngör T, Hartog N, Holland SM, Kohn DB, Malech HL, Markert ML, Weinacht KG, Villa A, Seet CS, Montel-Hagen A, Crooks GM, Notarangelo LD. PMID: 32556283; PMCID: PMC7322962.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCells
    28. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020 06 04; 135(23):2094-2105. Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, Bleesing JJ, Edwards S, Dvorak CC, Chaudhury S, Prockop SE, Quinones R, Goldman FD, Quigg TC, Chandrakasan S, Smith AR, Parikh S, Dávila Saldaña BJ, Thakar MS, Phelan R, Shenoy S, Forbes LR, Martinez C, Chellapandian D, Shereck E, Miller HK, Kapoor N, Barnum JL, Chong H, Shyr DC, Chen K, Abu-Arja R, Shah AJ, Weinacht KG, Moore TB, Joshi A, DeSantes KB, Gillio AP, Cuvelier GDE, Keller MD, Rozmus J, Torgerson T, Pulsipher MA, Haddad E, Sullivan KE, Logan BR, Kohn DB, Puck JM, Notarangelo LD, Pai SY, Rawlings DJ, Cowan MJ. PMID: 32268350; PMCID: PMC7273831.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCells
    29. Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences. Mol Ther Methods Clin Dev. 2020 Jun 12; 17:999-1013. Morgan RA, Ma F, Unti MJ, Brown D, Ayoub PG, Tam C, Lathrop L, Aleshe B, Kurita R, Nakamura Y, Senadheera S, Wong RL, Hollis RP, Pellegrini M, Kohn DB. PMID: 32426415; PMCID: PMC7225380.
      View in: PubMed   Mentions: 2  
    30. Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey. Front Immunol. 2020; 11:239. Chan AY, Leiding JW, Liu X, Logan BR, Burroughs LM, Allenspach EJ, Skoda-Smith S, Uzel G, Notarangelo LD, Slatter M, Gennery AR, Smith AR, Pai SY, Jordan MB, Marsh RA, Cowan MJ, Dvorak CC, Craddock JA, Prockop SE, Chandrakasan S, Kapoor N, Buckley RH, Parikh S, Chellapandian D, Oshrine BR, Bednarski JJ, Cooper MA, Shenoy S, Davila Saldana BJ, Forbes LR, Martinez C, Haddad E, Shyr DC, Chen K, Sullivan KE, Heimall J, Wright N, Bhatia M, Cuvelier GDE, Goldman FD, Meyts I, Miller HK, Seidel MG, Vander Lugt MT, Bacchetta R, Weinacht KG, Andolina JR, Caywood E, Chong H, de la Morena MT, Aquino VM, Shereck E, Walter JE, Dorsey MJ, Seroogy CM, Griffith LM, Kohn DB, Puck JM, Pulsipher MA, Torgerson TR. PMID: 32153572; PMCID: PMC7046837.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansAnimalsCells
    31. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med. 2020 02; 26(2):200-206. Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ, Net4CGD consortium. PMID: 31988463; PMCID: PMC7115833.
      View in: PubMed   Mentions: 40     Fields:    Translation:HumansCellsCTClinical Trials
    32. End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings. Blood Adv. 2019 12 10; 3(23):4002-4020. Farrell AT, Panepinto J, Desai AA, Kassim AA, Lebensburger J, Walters MC, Bauer DE, Blaylark RM, DiMichele DM, Gladwin MT, Green NS, Hassell K, Kato GJ, Klings ES, Kohn DB, Krishnamurti L, Little J, Makani J, Malik P, McGann PT, Minniti C, Morris CR, Odame I, Oneal PA, Setse R, Sharma P, Shenoy S. PMID: 31809537; PMCID: PMC6963248.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    33. Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice. Mol Ther Methods Clin Dev. 2020 Mar 13; 16:78-93. Carbonaro-Sarracino DA, Tarantal AF, Lee CCI, Kaufman ML, Wandro S, Jin X, Martinez M, Clark DN, Chun K, Koziol C, Hardee CL, Wang X, Kohn DB. PMID: 31871959; PMCID: PMC6909201.
      View in: PubMed   Mentions: 4  
    34. Improved Titer and Gene Transfer by Lentiviral Vectors Using Novel, Small β-Globin Locus Control Region Elements. Mol Ther. 2020 01 08; 28(1):328-340. Morgan RA, Unti MJ, Aleshe B, Brown D, Osborne KS, Koziol C, Ayoub PG, Smith OB, O'Brien R, Tam C, Miyahira E, Ruiz M, Quintos JP, Senadheera S, Hollis RP, Kohn DB. PMID: 31628051; PMCID: PMC6953778.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansAnimalsCells
    35. Development of Hematopoietic Stem Cell-Engineered Invariant Natural Killer T Cell Therapy for Cancer. Cell Stem Cell. 2019 10 03; 25(4):542-557.e9. Zhu Y, Smith DJ, Zhou Y, Li YR, Yu J, Lee D, Wang YC, Di Biase S, Wang X, Hardoy C, Ku J, Tsao T, Lin LJ, Pham AT, Moon H, McLaughlin J, Cheng D, Hollis RP, Campo-Fernandez B, Urbinati F, Wei L, Pang L, Rezek V, Berent-Maoz B, Macabali MH, Gjertson D, Wang X, Galic Z, Kitchen SG, An DS, Hu-Lieskovan S, Kaplan-Lefko PJ, De Oliveira SN, Seet CS, Larson SM, Forman SJ, Heath JR, Zack JA, Crooks GM, Radu CG, Ribas A, Kohn DB, Witte ON, Yang L. PMID: 31495780; PMCID: PMC7018522.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansAnimalsCells
    36. Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT. J Clin Immunol. 2019 10; 39(7):653-667. Marsh RA, Leiding JW, Logan BR, Griffith LM, Arnold DE, Haddad E, Falcone EL, Yin Z, Patel K, Arbuckle E, Bleesing JJ, Sullivan KE, Heimall J, Burroughs LM, Skoda-Smith S, Chandrakasan S, Yu LC, Oshrine BR, Cuvelier GDE, Thakar MS, Chen K, Teira P, Shenoy S, Phelan R, Forbes LR, Chellapandian D, Dávila Saldaña BJ, Shah AJ, Weinacht KG, Joshi A, Boulad F, Quigg TC, Dvorak CC, Grossman D, Torgerson T, Graham P, Prasad V, Knutsen A, Chong H, Miller H, de la Morena MT, DeSantes K, Cowan MJ, Notarangelo LD, Kohn DB, Stenger E, Pai SY, Routes JM, Puck JM, Kapoor N, Pulsipher MA, Malech HL, Parikh S, Kang EM, submitted on behalf of the Primary Immune Deficiency Treatment Consortium. PMID: 31376032; PMCID: PMC6754755.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
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    145. Improving cellular therapy for primary immune deficiency diseases: recognition, diagnosis, and management. J Allergy Clin Immunol. 2009 Dec; 124(6):1152-60.e12. Griffith LM, Cowan MJ, Notarangelo LD, Puck JM, Buckley RH, Candotti F, Conley ME, Fleisher TA, Gaspar HB, Kohn DB, Ochs HD, O'Reilly RJ, Rizzo JD, Roifman CM, Small TN, Shearer WT, Workshop Participants. PMID: 20004776; PMCID: PMC2831471.
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    146. Stable transgene expression in primitive human CD34+ hematopoietic stem/progenitor cells, using the Sleeping Beauty transposon system. Hum Gene Ther. 2009 Dec; 20(12):1607-26. Sumiyoshi T, Holt NG, Hollis RP, Ge S, Cannon PM, Crooks GM, Kohn DB. PMID: 19689196; PMCID: PMC4094131.
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    147. Preloading potential of retroviral vectors is packaging cell clone dependent and centrifugation onto CH-296 ensures highest transduction efficiency. Hum Gene Ther. 2009 Apr; 20(4):337-49. Sondergaard CS, Haldrup C, Beer C, Andersen B, Kohn DB, Pedersen L. PMID: 19281432.
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    148. Gene therapy fulfilling its promise. N Engl J Med. 2009 Jan 29; 360(5):518-21. Kohn DB, Candotti F. PMID: 19179320.
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    149. Pre- and post-natal treatment of hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer. 2009 Jan; 52(1):139-42. Shah AJ, Kapoor N, Cooper RM, Crooks GM, Lenarsky C, Abdel-Azim H, Wu SQ, Wilson K, Weinberg KI, Parkman R, Kohn DB. PMID: 18819128; PMCID: PMC2592085.
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    150. Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. J Allergy Clin Immunol. 2008 Dec; 122(6):1087-96. Griffith LM, Cowan MJ, Kohn DB, Notarangelo LD, Puck JM, Schultz KR, Buckley RH, Eapen M, Kamani NR, O'Reilly RJ, Parkman R, Roifman CM, Sullivan KE, Filipovich AH, Fleisher TA, Shearer WT. PMID: 18992926; PMCID: PMC3357108.
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    151. Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). J Clin Immunol. 2009 Mar; 29(2):231-7. Lin M, Epport K, Azen C, Parkman R, Kohn DB, Shah AJ. PMID: 18807155; PMCID: PMC7102058.
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    152. Clinical and genetic heterogeneity in Omenn syndrome and severe combined immune deficiency. Pediatr Transplant. 2009 Mar; 13(2):244-50. Gruber TA, Shah AJ, Hernandez M, Crooks GM, Abdel-Azim H, Gupta S, McKnight S, White D, Kapoor N, Kohn DB. PMID: 18822103.
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    153. CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model. J Immunol. 2008 Jul 15; 181(2):1063-70. Chhabra A, Yang L, Wang P, Comin-Anduix B, Das R, Chakraborty NG, Ray S, Mehrotra S, Yang H, Hardee CL, Hollis R, Dorsky DI, Koya R, Kohn DB, Ribas A, Economou JS, Baltimore D, Mukherji B. PMID: 18606658; PMCID: PMC2715965.
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    154. Progressive declines in neurocognitive function among survivors of hematopoietic stem cell transplantation for pediatric hematologic malignancies. J Pediatr Hematol Oncol. 2008 Jun; 30(6):411-8. Shah AJ, Epport K, Azen C, Killen R, Wilson K, De Clerck D, Crooks G, Kapoor N, Kohn DB, Parkman R, Weinberg KI. PMID: 18525456.
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    155. In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther. 2008 Jul; 16(7):1308-15. Bauer G, Dao MA, Case SS, Meyerrose T, Wirthlin L, Zhou P, Wang X, Herrbrich P, Arevalo J, Csik S, Skelton DC, Walker J, Pepper K, Kohn DB, Nolta JA. PMID: 18461052; PMCID: PMC3013368.
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    156. Redirecting specificity of T-cell populations for CD19 using the Sleeping Beauty system. Cancer Res. 2008 Apr 15; 68(8):2961-71. Singh H, Manuri PR, Olivares S, Dara N, Dawson MJ, Huls H, Hackett PB, Kohn DB, Shpall EJ, Champlin RE, Cooper LJ. PMID: 18413766; PMCID: PMC2424272.
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    157. Neonatal bone marrow transplantation of ADA-deficient SCID mice results in immunologic reconstitution despite low levels of engraftment and an absence of selective donor T lymphoid expansion. Blood. 2008 Jun 15; 111(12):5745-54. Carbonaro DA, Jin X, Cotoi D, Mi T, Yu XJ, Skelton DC, Dorey F, Kellems RE, Blackburn MR, Kohn DB. PMID: 18356486; PMCID: PMC2424166.
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    158. Expansion of multipotent and lymphoid-committed human progenitors through intracellular dimerization of Mpl. Blood. 2008 Apr 15; 111(8):4064-74. Abdel-Azim H, Zhu Y, Hollis R, Wang X, Ge S, Hao QL, Smbatyan G, Kohn DB, Rosol M, Crooks GM. PMID: 18174381; PMCID: PMC2288719.
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    159. Human hematopoietic cell culture, transduction, and analyses. Curr Protoc Hum Genet. 2008 Jan; Chapter 13:Unit 13.7. Bonde J, Wirthlin L, Kohn DB, Nolta JA. PMID: 18428422.
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    160. Genetic therapies against HIV. Nat Biotechnol. 2007 Dec; 25(12):1444-54. Rossi JJ, June CH, Kohn DB. PMID: 18066041; PMCID: PMC4539027.
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    161. Manipulation of OCT4 levels in human embryonic stem cells results in induction of differential cell types. Exp Biol Med (Maywood). 2007 Nov; 232(10):1368-80. Rodriguez RT, Velkey JM, Lutzko C, Seerke R, Kohn DB, O'Shea KS, Firpo MT. PMID: 17959850.
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    162. Foamy virus vectors expressing anti-HIV transgenes efficiently block HIV-1 replication. Mol Ther. 2008 Jan; 16(1):46-51. Taylor JA, Vojtech L, Bahner I, Kohn DB, Laer DV, Russell DW, Richard RE. PMID: 17955023.
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    163. Preferential association of prostate cancer cells expressing prostate specific membrane antigen to bone marrow matrix. Int J Oncol. 2007 Apr; 30(4):899-904. Barwe SP, Maul RS, Christiansen JJ, Anilkumar G, Cooper CR, Kohn DB, Rajasekaran AK. PMID: 17332929.
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    164. The effects of Campath 1H upon graft-versus-host disease, infection, relapse, and immune reconstitution in recipients of pediatric unrelated transplants. Biol Blood Marrow Transplant. 2007 May; 13(5):584-93. Shah AJ, Kapoor N, Crooks GM, Weinberg KI, Azim HA, Killen R, Kuo L, Rushing T, Kohn DB, Parkman R. PMID: 17448918.
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    165. Effective suicide gene therapy for leukemia in a model of insertional oncogenesis in mice. Mol Ther. 2007 Jan; 15(1):183-92. Blumenthal M, Skelton D, Pepper KA, Jahn T, Methangkool E, Kohn DB. PMID: 17164790.
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    166. Lentiviral vector transduction of a dominant-negative Rev gene into human CD34+ hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Mol Ther. 2007 Jan; 15(1):76-85. Bahner I, Sumiyoshi T, Kagoda M, Swartout R, Peterson D, Pepper K, Dorey F, Reiser J, Kohn DB. PMID: 17164778.
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    167. Lentiviral vectors ready for prime-time. Nat Biotechnol. 2007 Jan; 25(1):65-6. Kohn DB. PMID: 17211402.
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    168. Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Exp Hematol. 2006 Oct; 34(10):1333-43. Hollis RP, Nightingale SJ, Wang X, Pepper KA, Yu XJ, Barsky L, Crooks GM, Kohn DB. PMID: 16982326.
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    169. Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report. Blood. 2007 Jan 15; 109(2):503-6. Engel BC, Podsakoff GM, Ireland JL, Smogorzewska EM, Carbonaro DA, Wilson K, Shah A, Kapoor N, Sweeney M, Borchert M, Crooks GM, Weinberg KI, Parkman R, Rosenblatt HM, Wu SQ, Hershfield MS, Candotti F, Kohn DB. PMID: 16973956; PMCID: PMC1785101.
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    170. Association of prostate-specific membrane antigen with caveolin-1 and its caveolae-dependent internalization in microvascular endothelial cells: implications for targeting to tumor vasculature. Microvasc Res. 2006 Jul-Sep; 72(1-2):54-61. Anilkumar G, Barwe SP, Christiansen JJ, Rajasekaran SA, Kohn DB, Rajasekaran AK. PMID: 16713605.
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    171. In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement therapy for ADA deficiency. Mol Ther. 2006 Jun; 13(6):1110-20. Carbonaro DA, Jin X, Petersen D, Wang X, Dorey F, Kil KS, Aldrich M, Blackburn MR, Kellems RE, Kohn DB. PMID: 16651028.
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    172. Transient gene expression by nonintegrating lentiviral vectors. Mol Ther. 2006 Jun; 13(6):1121-32. Nightingale SJ, Hollis RP, Pepper KA, Petersen D, Yu XJ, Yang C, Bahner I, Kohn DB. PMID: 16556511.
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    173. Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Exp Hematol. 2006 Mar; 34(3):369-81. Kahl CA, Tarantal AF, Lee CI, Jimenez DF, Choi C, Pepper K, Petersen D, Fletcher MD, Leapley AC, Fisher J, Burns TS, Ultsch MN, Dorey FJ, Kohn DB. PMID: 16543071.
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    174. Specific and stable gene transfer to human embryonic stem cells using pseudotyped lentiviral vectors. Stem Cells Dev. 2006 Feb; 15(1):109-17. Jang JE, Shaw K, Yu XJ, Petersen D, Pepper K, Lutzko C, Kohn DB. PMID: 16522168.
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    175. Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein. J Virol. 2006 Feb; 80(4):1863-73. Unwalla HJ, Li HT, Bahner I, Li MJ, Kohn D, Rossi JJ. PMID: 16439542; PMCID: PMC1367144.
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    176. Perspectives on gene therapy for immune deficiencies. Biol Blood Marrow Transplant. 2005 Dec; 11(12):972-6. Podsakoff GM, Engel BC, Kohn DB. PMID: 16338618.
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    177. Tolerance induction by lentiviral gene therapy with a nonmyeloablative regimen. Blood. 2006 Mar 15; 107(6):2286-93. Mitsuhashi N, Fischer-Lougheed J, Shulkin I, Kleihauer A, Kohn DB, Weinberg KI, Starnes VA, Kearns-Jonker M. PMID: 16291598.
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    178. Recombinant murine interleukin-12 elicits potent antileukemic immune responses in a murine model of Philadelphia chromosome-positive acute lymphoblastic leukemia. Cancer Gene Ther. 2005 Oct; 12(10):818-24. Gruber TA, Skelton DC, Kohn DB. PMID: 15877085.
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    179. Successful hematopoietic stem cell transplantation for Niemann-Pick disease type B. Pediatrics. 2005 Oct; 116(4):1022-5. Shah AJ, Kapoor N, Crooks GM, Parkman R, Weinberg KI, Wilson K, Kohn DB. PMID: 16199719.
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    180. Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Clin Immunol. 2005 Nov; 117(2):133-43. Chan B, Wara D, Bastian J, Hershfield MS, Bohnsack J, Azen CG, Parkman R, Weinberg K, Kohn DB. PMID: 16112907.
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    181. Selective survival of peripheral blood lymphocytes in children with HIV-1 following delivery of an anti-HIV gene to bone marrow CD34(+) cells. Mol Ther. 2005 Jul; 12(1):77-86. Podsakoff GM, Engel BC, Carbonaro DA, Choi C, Smogorzewska EM, Bauer G, Selander D, Csik S, Wilson K, Betts MR, Koup RA, Nabel GJ, Bishop K, King S, Schmidt M, von Kalle C, Church JA, Kohn DB. PMID: 15963923.
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    182. Intrapulmonary and intramyocardial gene transfer in rhesus monkeys (Macaca mulatta): safety and efficiency of HIV-1-derived lentiviral vectors for fetal gene delivery. Mol Ther. 2005 Jul; 12(1):87-98. Tarantal AF, McDonald RJ, Jimenez DF, Lee CC, O'Shea CE, Leapley AC, Won RH, Plopper CG, Lutzko C, Kohn DB. PMID: 15963924.
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    183. Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector. Mol Ther. 2005 May; 11(5):776-89. Kobayashi H, Carbonaro D, Pepper K, Petersen D, Ge S, Jackson H, Shimada H, Moats R, Kohn DB. PMID: 15851016.
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    184. Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta). Hum Gene Ther. 2005 Apr; 16(4):417-25. Lee CC, Jimenez DF, Kohn DB, Tarantal AF. PMID: 15871673.
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    185. Factors influencing the titer and infectivity of lentiviral vectors. Hum Gene Ther. 2004 Oct; 15(10):976-88. Logan AC, Nightingale SJ, Haas DL, Cho GJ, Pepper KA, Kohn DB. PMID: 15585113.
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    186. Integrated self-inactivating lentiviral vectors produce full-length genomic transcripts competent for encapsidation and integration. J Virol. 2004 Aug; 78(16):8421-36. Logan AC, Haas DL, Kafri T, Kohn DB. PMID: 15280451; PMCID: PMC479072.
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    187. Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol Genet Metab. 2004 Aug; 82(4):286-95. Zheng Y, Ryazantsev S, Ohmi K, Zhao HZ, Rozengurt N, Kohn DB, Neufeld EF. PMID: 15308126.
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    188. Use of lentiviral vectors to induce long-term tolerance to gal(+) heart grafts. Transplantation. 2004 Jun 15; 77(11):1748-54. Kearns-Jonker M, Fischer-Lougheed J, Shulkin I, Kleihauer A, Mitsuhashi N, Kohn DB, Weinberg K, D'Apice AJ, Starnes VA, Cramer DV. PMID: 15201677.
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    189. Simian immunodeficiency virus infection of hematopoietic stem cells and bone marrow stromal cells. J Acquir Immune Defic Syndr. 2004 May 01; 36(1):553-61. Lee CI, Cowan MJ, Kohn DB, Tarantal AF. PMID: 15097297.
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    190. Busulfan and cyclophosphamide as a conditioning regimen for pediatric acute lymphoblastic leukemia patients undergoing bone marrow transplantation. J Pediatr Hematol Oncol. 2004 Feb; 26(2):91-7. Shah AJ, Lenarsky C, Kapoor N, Crooks GM, Kohn DB, Parkman R, Epport K, Wilson K, Weinberg K. PMID: 14767194.
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    191. Morphological analysis and lentiviral transduction of fetal monkey bone marrow-derived mesenchymal stem cells. Mol Ther. 2004 Jan; 9(1):112-23. Lee CI, Kohn DB, Ekert JE, Tarantal AF. PMID: 14741784.
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    192. Update on gene therapy of inherited immune deficiencies. Curr Opin Mol Ther. 2003 Oct; 5(5):503-7. Engel BC, Kohn DB, Podsakoff GM. PMID: 14601519.
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    193. Transduction of green fluorescent protein increased oxidative stress and enhanced sensitivity to cytotoxic drugs in neuroblastoma cell lines. Mol Cancer Ther. 2003 Sep; 2(9):911-7. Goto H, Yang B, Petersen D, Pepper KA, Alfaro PA, Kohn DB, Reynolds CP. PMID: 14555710.
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    194. The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells. J Virol. 2003 Sep; 77(17):9439-50. Haas DL, Lutzko C, Logan AC, Cho GJ, Skelton D, Jin Yu X, Pepper KA, Kohn DB. PMID: 12915559; PMCID: PMC187403.
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    195. Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Blood. 2003 Nov 15; 102(10):3478-82. Wang X, Rosol M, Ge S, Peterson D, McNamara G, Pollack H, Kohn DB, Nelson MD, Crooks GM. PMID: 12946998.
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    196. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther. 2003 Aug; 8(2):180-7. Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F, Tisdale J, Riviére I, Blau CA, Richard RE, Sorrentino B, Nolta J, Malech H, Brenner M, Cornetta K, Cavagnaro J, High K, Glorioso J, American Society of Gene Therapy (ASGT). PMID: 12907140.
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    197. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow. Mol Genet Metab. 2003 Aug; 79(4):233-44. Zheng Y, Rozengurt N, Ryazantsev S, Kohn DB, Satake N, Neufeld EF. PMID: 12948739.
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    198. Scaffold attachment region-containing retrovirus vectors improve long-term proviral expression after transplantation of GFP-modified CD34+ baboon repopulating cells. Blood. 2003 Nov 01; 102(9):3117-9. Kurre P, Morris J, Thomasson B, Kohn DB, Kiem HP. PMID: 12869503.
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    199. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer. 2003 Jul; 3(7):477-88. Kohn DB, Sadelain M, Glorioso JC. PMID: 12835668.
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    200. Letter to the editors of Nature from the American Society of Gene Therapy (ASGT) and the European Society of Gene Therapy (ESGT). J Gene Med. 2003 Jul; 5(7):641. Kohn DB, Gänsbacher B. PMID: 12825204.
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    201. Lentivirus vectors incorporating the immunoglobulin heavy chain enhancer and matrix attachment regions provide position-independent expression in B lymphocytes. J Virol. 2003 Jul; 77(13):7341-51. Lutzko C, Senadheera D, Skelton D, Petersen D, Kohn DB. PMID: 12805432; PMCID: PMC164820.
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    202. Efficient characterization of retro-, lenti-, and foamyvector-transduced cell populations by high-accuracy insertion site sequencing. Ann N Y Acad Sci. 2003 May; 996:112-21. Schmidt M, Glimm H, Wissler M, Hoffmann G, Olsson K, Sellers S, Carbonaro D, Tisdale JF, Leurs C, Hanenberg H, Dunbar CE, Kiem HP, Karlsson S, Kohn DB, Williams D, Von Kalle C. PMID: 12799289.
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    203. Effects of the negative control region on expression from retroviral LTR. Mol Ther. 2003 Apr; 7(4):438-40. Wang L, Haas D, Halene S, Kohn DB. PMID: 12727105.
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    204. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat Med. 2003 Apr; 9(4):463-8. Schmidt M, Carbonaro DA, Speckmann C, Wissler M, Bohnsack J, Elder M, Aronow BJ, Nolta JA, Kohn DB, von Kalle C. PMID: 12640448.
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    205. [Transplantation of genetically modified cells in the treatment of children with SCID: great hopes and recent disappointments]. Med Wieku Rozwoj. 2003 Jan-Mar; 7(1):27-34. Smogorzewska EM, Weinberg KI, Kohn DB. PMID: 13130167.
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    206. Gene therapy for inborn and acquired immune deficiency disorders. Acta Haematol. 2003; 110(2-3):60-70. Engel BC, Kohn DB. PMID: 14583666.
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    207. Marrow-derived cells as vehicles for delivery of gene therapy to pulmonary epithelium. Am J Respir Cell Mol Biol. 2002 Dec; 27(6):645-51. Grove JE, Lutzko C, Priller J, Henegariu O, Theise ND, Kohn DB, Krause DS. PMID: 12444022.
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    208. Expression from second-generation feline immunodeficiency virus vectors is impaired in human hematopoietic cells. Mol Ther. 2002 Nov; 6(5):645-52. Price MA, Case SS, Carbonaro DA, Yu XJ, Petersen D, Sabo KM, Curran MA, Engel BC, Margarian H, Abkowitz JL, Nolan GP, Kohn DB, Crooks GM. PMID: 12409263.
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    209. In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression. Hum Gene Ther. 2002 Oct 10; 13(15):1809-20. Meertens L, Zhao Y, Rosic-Kablar S, Li L, Chan K, Dobson H, Gartley C, Lutzko C, Hopwood J, Kohn D, Kruth S, Hough MR, Dubé ID. PMID: 12396614.
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    210. Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol. 2002 Oct; 13(5):429-36. Logan AC, Lutzko C, Kohn DB. PMID: 12459333.
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    211. Gene therapy of RAG-2-/- mice: sustained correction of the immunodeficiency. Blood. 2002 Dec 01; 100(12):3942-9. Yates F, Malassis-Séris M, Stockholm D, Bouneaud C, Larousserie F, Noguiez-Hellin P, Danos O, Kohn DB, Fischer A, de Villartay JP, Cavazzana-Calvo M. PMID: 12393742.
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    212. Kinetics of fluorescence expression in nonhuman primates transplanted with GFP retrovirus-modified CD34 cells. Mol Ther. 2002 Jul; 6(1):83-90. Kurre P, Morris J, Andrews RG, Kohn DB, Kiem HP. PMID: 12095307.
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    213. Adenosine deaminase gene therapy protocol revisited. Mol Ther. 2002 Feb; 5(2):96-7. Kohn DB. PMID: 11829515.
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    214. Requirement for NK cells in CD40 ligand-mediated rejection of Philadelphia chromosome-positive acute lymphoblastic leukemia cells. J Immunol. 2002 Jan 01; 168(1):73-80. Gruber TA, Skelton DC, Kohn DB. PMID: 11751948.
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    215. Second hematopoietic stem cell transplantation in pediatric patients: overall survival and long-term follow-up. Biol Blood Marrow Transplant. 2002; 8(4):221-8. Shah AJ, Kapoor N, Weinberg KI, Crooks GM, Kohn DB, Lenarsky C, Kaufman F, Epport K, Wilson K, Parkman R. PMID: 12014811.
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