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. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. Blood. 2022 Aug 18; 140(7):685-705. Cuvelier GDE, Logan BR, Prockop SE, Buckley RH, Kuo CY, Griffith LM, Liu X, Yip A, Hershfield MS, Ayoub PG, Moore TB, Dorsey MJ, O'Reilly RJ, Kapoor N, Pai SY, Kapadia M, Ebens CL, Forbes Satter LR, Burroughs LM, Petrovic A, Chellapandian D, Heimall J, Shyr DC, Rayes A, Bednarski JJ, Chandra S, Chandrakasan S, Gillio AP, Madden L, Quigg TC, Caywood EH, Dávila Saldaña BJ, DeSantes K, Eissa H, Goldman FD, Rozmus J, Shah AJ, Vander Lugt MT, Thakar MS, Parrott RE, Martinez C, Leiding JW, Torgerson TR, Pulsipher MA, Notarangelo LD, Cowan MJ, Dvorak CC, Haddad E, Puck JM, Kohn DB. PMID: 35671392.
      View in: PubMed   Mentions:    Fields:    
    2. Improving Lentiviral Transduction of Human Adipose-Derived Mesenchymal Stem Cells. Hum Gene Ther. 2022 Jul 20. Collon K, Gallo M, Bell J, Chang S, Rodman J, Sugiyama O, Kohn DB, Lieberman JR. PMID: 35859364.
      View in: PubMed   Mentions:    Fields:    
    3. Granulocyte Transfusions in Patients with Chronic Granulomatous Disease Undergoing Hematopoietic Cell Transplantation or Gene Therapy. J Clin Immunol. 2022 07; 42(5):1026-1035. 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:    Fields:    
    4. 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     Fields:    Translation:HumansCellsPHPublic Health
    5. 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: 1  
    6. 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: 7     Fields:    Translation:HumansAnimalsCells
    7. 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: 4     Fields:    Translation:HumansCTClinical Trials
    8. 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: 1     Fields:    Translation:HumansAnimals
    9. 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: 2  
    10. 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:    Translation:Humans
    11. 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: 1     Fields:    Translation:Animals
    12. 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: 21     Fields:    Translation:HumansCellsCTClinical Trials
    13. 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: 1     Fields:    Translation:HumansCells
    14. 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: 26     Fields:    Translation:HumansAnimalsCells
    15. Gene Therapies for Primary Immune Deficiencies. Front Immunol. 2021; 12:648951. Kohn LA, Kohn DB. PMID: 33717203; PMCID: PMC7946985.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansCells
    16. 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  
    17. 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:    
    18. 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: 3     Fields:    Translation:HumansCells
    19. 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: 1  
    20. 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: 4     Fields:    Translation:HumansCells
    21. 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:    
    22. Supramolecular nanosubstrate-mediated delivery system enables CRISPR-Cas9 knockin of hemoglobin beta gene for hemoglobinopathies. Sci Adv. 2020 10; 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: 8     Fields:    Translation:Animals
    23. 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: 2     Fields:    Translation:HumansPHPublic Health
    24. 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
    25. 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: 3     Fields:    Translation:HumansAnimals
    26. 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: 1     Fields:    Translation:Humans
    27. 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: 3     Fields:    Translation:Humans
    28. 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: 10     Fields:    Translation:HumansAnimalsCells
    29. 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: 22     Fields:    Translation:HumansCells
    30. 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: 24     Fields:    Translation:HumansCells
    31. 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: 4  
    32. 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: 13     Fields:    Translation:HumansAnimalsCells
    33. 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: 62     Fields:    Translation:HumansCellsCTClinical Trials
    34. 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: 6     Fields:    Translation:Humans
    35. 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: 5  
    36. 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: 15     Fields:    Translation:HumansAnimalsCells
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    47. Improving Gene Editing Outcomes in Human Hematopoietic Stem and Progenitor Cells by Temporal Control of DNA Repair. Stem Cells. 2019 02; 37(2):284-294. Lomova A, Clark DN, Campo-Fernandez B, Flores-Bjurström C, Kaufman ML, Fitz-Gibbon S, Wang X, Miyahira EY, Brown D, DeWitt MA, Corn JE, Hollis RP, Romero Z, Kohn DB. PMID: 30372555; PMCID: PMC6368869.
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    48. IND-Enabling Studies for a Clinical Trial to Genetically Program a Persistent Cancer-Targeted Immune System. Clin Cancer Res. 2019 02 01; 25(3):1000-1011. Puig-Saus C, Parisi G, Garcia-Diaz A, Krystofinski PE, Sandoval S, Zhang R, Champhekar AS, McCabe J, Cheung-Lau GC, Truong NA, Vega-Crespo A, Komenan MDS, Pang J, Macabali MH, Saco JD, Goodwin JL, Bolon B, Seet CS, Montel-Hagen A, Crooks GM, Hollis RP, Campo-Fernandez B, Bischof D, Cornetta K, Gschweng EH, Adelson C, Nguyen A, Yang L, Witte ON, Baltimore D, Comin-Anduix B, Kohn DB, Wang X, Cabrera P, Kaplan-Lefko PJ, Berent-Maoz B, Ribas A. PMID: 30409823; PMCID: PMC6359988.
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