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. Balancing efficacy and safety in lentiviral vector-mediated hematopoietic stem cell gene therapy. Mol Ther. 2024 Dec 26. Montini E, Naldini L, Booth C, Kohn DB, Aiuti A. PMID: 39729983.
      View in: PubMed   Mentions:    Fields:    
    2. Myelodysplasia after Lentiviral Gene Therapy. N Engl J Med. 2024 Dec 19; 391(24):2382. Kohn DB, Booth C, Naldini L. PMID: 39693551.
      View in: PubMed   Mentions:    Fields:    
    3. Making Sense of Adenosine Deaminase Variants and Their Clinical Implications. J Allergy Clin Immunol. 2024 Nov 15. Grunebaum E, Loves R, Kohn DB. PMID: 39551441.
      View in: PubMed   Mentions:    Fields:    
    4. DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential. Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4):101334. Luthers CR, Ha SM, Mittelhauser A, Morselli M, Long JD, Kuo CY, Romero Z, Kohn DB. PMID: 39381161; PMCID: PMC11460252.
      View in: PubMed   Mentions:
    5. Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease. Mol Ther Methods Clin Dev. 2024 Dec 12; 32(4):101323. Ayoub PG, Gensheimer J, Lathrop L, Juett C, Quintos J, Tam K, Reid J, Ma F, Tam C, McAuley GE, Brown D, Wu X, Zhang R, Bradford K, Hollis RP, Crooks GM, Kohn DB. PMID: 39309261; PMCID: PMC11415656.
      View in: PubMed   Mentions:
    6. Enhancing pediatric access to cell and gene therapies. Nat Med. 2024 Jul; 30(7):1836-1846. Mackall CL, Bollard CM, Goodman N, Carr C, Gardner R, Rouce R, Sotillo E, Stoner R, Urnov FD, Wayne AS, Park J, Kohn DB. PMID: 38886624.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    7. Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method. Nat Biotechnol. 2024 May 14. Li YR, Zhou Y, Yu J, Kim YJ, Li M, Lee D, Zhou K, Chen Y, Zhu Y, Wang YC, Li Z, Yu Y, Dunn ZS, Guo W, Cen X, Husman T, Bajpai A, Kramer A, Wilson M, Fang Y, Huang J, Li S, Zhou Y, Zhang Y, Hahn Z, Zhu E, Ma F, Pan C, Lusis AJ, Zhou JJ, Seet CS, Kohn DB, Wang P, Zhou XJ, Pellegrini M, Puliafito BR, Larson SM, Yang L. PMID: 38744947.
      View in: PubMed   Mentions: 5     Fields:    
    8. A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease. Mol Ther Methods Clin Dev. 2024 Jun 13; 32(2):101254. Hart KL, Liu B, Brown D, Campo-Fernandez B, Tam K, Orr K, Hollis RP, Brendel C, Williams DA, Kohn DB. PMID: 38745893; PMCID: PMC11091523.
      View in: PubMed   Mentions:
    9. Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment. Nat Commun. 2024 Apr 18; 15(1):3258. Keller MD, Hanley PJ, Chi YY, Aguayo-Hiraldo P, Dvorak CC, Verneris MR, Kohn DB, Pai SY, Dávila Saldaña BJ, Hanisch B, Quigg TC, Adams RH, Dahlberg A, Chandrakasan S, Hasan H, Malvar J, Jensen-Wachspress MA, Lazarski CA, Sani G, Idso JM, Lang H, Chansky P, McCann CD, Tanna J, Abraham AA, Webb JL, Shibli A, Keating AK, Satwani P, Muranski P, Hall E, Eckrich MJ, Shereck E, Miller H, Mamcarz E, Agarwal R, De Oliveira SN, Vander Lugt MT, Ebens CL, Aquino VM, Bednarski JJ, Chu J, Parikh S, Whangbo J, Lionakis M, Zambidis ET, Gourdine E, Bollard CM, Pulsipher MA. PMID: 38637498; PMCID: PMC11026387.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCellsCTClinical Trials
    10. Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Clin Immunol. 2024 04; 261:109942. Abraham RS, Basu A, Heimall JR, Dunn E, Yip A, Kapadia M, Kapoor N, Satter LF, Buckley R, O'Reilly R, Cuvelier GDE, Chandra S, Bednarski J, Chaudhury S, Moore TB, Haines H, Dávila Saldaña BJ, Chellapandian D, Rayes A, Chen K, Caywood E, Chandrakasan S, Lugt MTV, Ebens C, Teira P, Shereck E, Miller H, Aquino V, Eissa H, Yu LC, Gillio A, Madden L, Knutsen A, Shah AJ, DeSantes K, Barnum J, Broglie L, Joshi AY, Kleiner G, Dara J, Prockop S, Martinez C, Mousallem T, Oved J, Burroughs L, Marsh R, Torgerson TR, Leiding JW, Pai SY, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Cowan MJ, Puck J, Dvorak CC, Haddad E. PMID: 38367737; PMCID: PMC11018339.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCellsPHPublic Health
    11. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study. J Allergy Clin Immunol. 2024 May; 153(5):1423-1431.e2. Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Deal CL, Kapoor N, Murguía-Favela L, Falcone EL, Prasad VK, Touzot F, Bleesing JJ, Chandrakasan S, Heimall JR, Bednarski JJ, Broglie LA, Chong HJ, Kapadia M, Prockop S, Dávila Saldaña BJ, Schaefer E, Bauchat AL, Teira P, Chandra S, Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Leiding JW. PMID: 38290608; PMCID: PMC11070290.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    12. What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity. J Allergy Clin Immunol Pract. 2024 May; 12(5):1139-1149. Mudde ACA, Kuo CY, Kohn DB, Booth C. PMID: 38246560.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimals
    13. Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD. Blood. 2023 12 14; 142(24):2105-2118. Leiding JW, Arnold DE, Parikh S, Logan B, Marsh RA, Griffith LM, Wu R, Kidd S, Mallhi K, Chellapandian D, Si Lim SJ, Grunebaum E, Falcone EL, Murguia-Favela L, Grossman D, Prasad VK, Heimall JR, Touzot F, Burroughs LM, Bleesing J, Kapoor N, Dara J, Williams O, Kapadia M, Oshrine BR, Bednarski JJ, Rayes A, Chong H, Cuvelier GDE, Forbes Satter LR, Martinez C, Vander Lugt MT, Yu LC, Chandrakasan S, Joshi A, Prockop SE, Dávila Saldaña BJ, Aquino V, Broglie LA, Ebens CL, Madden LM, DeSantes K, Milner J, Rangarajan HG, Shah AJ, Gillio AP, Knutsen AP, Miller HK, Moore TB, Graham P, Bauchat A, Bunin NJ, Teira P, Petrovic A, Chandra S, Abdel-Azim H, Dorsey MJ, Birbrayer O, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM. PMID: 37562003; PMCID: PMC10862239.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    14. Gene therapy for adenosine deaminase severe combined immune deficiency-An unexpected journey of four decades. Immunol Rev. 2024 Mar; 322(1):148-156. Kohn DB. PMID: 38033164.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    15. Successes and challenges in clinical gene therapy. Gene Ther. 2023 11; 30(10-11):738-746. Kohn DB, Chen YY, Spencer MJ. PMID: 37935854; PMCID: PMC10678346.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCells
    16. Busulfan and subsequent malignancy: An evidence-based risk assessment. Pediatr Blood Cancer. 2024 Jan; 71(1):e30738. Long-Boyle JR, Kohn DB, Shah AJ, Spencer SM, Sevilla J, Booth C, López Lorenzo JL, Nicoletti E, Shah A, Reatz M, Matos J, Schwartz JD. PMID: 37856098.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    17. Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study. J Allergy Clin Immunol. 2024 01; 153(1):287-296. Eissa H, Thakar MS, Shah AJ, Logan BR, Griffith LM, Dong H, Parrott RE, O'Reilly RJ, Dara J, Kapoor N, Forbes Satter L, Chandra S, Kapadia M, Chandrakasan S, Knutsen A, Jyonouchi SC, Molinari L, Rayes A, Ebens CL, Teira P, Dávila Saldaña BJ, Burroughs LM, Chaudhury S, Chellapandian D, Gillio AP, Goldman F, Malech HL, DeSantes K, Cuvelier GDE, Rozmus J, Quinones R, Yu LC, Broglie L, Aquino V, Shereck E, Moore TB, Vander Lugt MT, Mousallem TI, Oved JH, Dorsey M, Abdel-Azim H, Martinez C, Bleesing JH, Prockop S, Kohn DB, Bednarski JJ, Leiding J, Marsh RA, Torgerson T, Notarangelo LD, Pai SY, Pulsipher MA, Puck JM, Dvorak CC, Haddad E, Buckley RH, Cowan MJ, Heimall J. PMID: 37793572; PMCID: PMC11294800.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    18. Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease. J Allergy Clin Immunol. 2023 12; 152(6):1619-1633.e11. Chandrasekaran P, Han Y, Zerbe CS, Heller T, DeRavin SS, Kreuzberg SA, Marciano BE, Siu Y, Jones DR, Abraham RS, Stephens MC, Tsou AM, Snapper S, Conlan S, Subramanian P, Quinones M, Grou C, Calderon V, Deming C, Leiding JW, Arnold DE, Logan BR, Griffith LM, Petrovic A, Mousallem TI, Kapoor N, Heimall JR, Barnum JL, Kapadia M, Wright N, Rayes A, Chandra S, Broglie LA, Chellapandian D, Deal CL, Grunebaum E, Lim SS, Mallhi K, Marsh RA, Murguia-Favela L, Parikh S, Touzot F, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Kang EM, Malech HL, Segre JA, Bryant CE, Holland SM, Falcone EL. PMID: 37659505; PMCID: PMC11279821.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    19. Fluorinated Silane-Modified Filtroporation Devices Enable Gene Knockout in Human Hematopoietic Stem and Progenitor Cells. ACS Appl Mater Interfaces. 2023 Sep 06; 15(35):41299-41309. Frost IM, Mendoza AM, Chiou TT, Kim P, Aizenberg J, Kohn DB, De Oliveira SN, Weiss PS, Jonas SJ. PMID: 37616579; PMCID: PMC10485797.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    20. Biodistribution of lentiviral transduced adipose-derived stem cells for "ex-vivo" regional gene therapy for bone repair. Gene Ther. 2023 12; 30(12):826-834. Bell JA, Collon K, Mayfield C, Gallo MC, Chang SW, Sugiyama O, Tang AH, Hollis R, Chopra S, Kohn DB, Lieberman JR. PMID: 37568039.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimalsCells
    21. Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium. Lancet. 2023 07 08; 402(10396):129-140. Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ, O'Reilly RJ, Kapoor N, Satter LF, Pai SY, Heimall J, Chandra S, Ebens CL, Chellapandian D, Williams O, Burroughs LM, Saldana BD, Rayes A, Madden LM, Chandrakasan S, Bednarski JJ, DeSantes KB, Cuvelier GDE, Teira P, Gillio AP, Eissa H, Knutsen AP, Goldman FD, Aquino VM, Shereck EB, Moore TB, Caywood EH, Lugt MTV, Rozmus J, Broglie L, Yu LC, Shah AJ, Andolina JR, Liu X, Parrott RE, Dara J, Prockop S, Martinez CA, Kapadia M, Jyonouchi SC, Sullivan KE, Bleesing JJ, Chaudhury S, Petrovic A, Keller MD, Quigg TC, Parikh S, Shenoy S, Seroogy C, Rubin T, Decaluwe H, Routes JM, Torgerson TR, Leiding JW, Pulsipher MA, Kohn DB, Griffith LM, Haddad E, Dvorak CC, Notarangelo LD. PMID: 37352885; PMCID: PMC10386791.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansPHPublic Health
    22. Human T cell generation is restored in CD3δ severe combined immunodeficiency through adenine base editing. Cell. 2023 03 30; 186(7):1398-1416.e23. McAuley GE, Yiu G, Chang PC, Newby GA, Campo-Fernandez B, Fitz-Gibbon ST, Wu X, Kang SL, Garibay A, Butler J, Christian V, Wong RL, Everette KA, Azzun A, Gelfer H, Seet CS, Narendran A, Murguia-Favela L, Romero Z, Wright N, Liu DR, Crooks GM, Kohn DB. PMID: 36944331; PMCID: PMC10876291.
      View in: PubMed   Mentions: 26     Fields:    Translation:HumansAnimalsCells
    23. β-Thalassemia: all about that base, no cutting. Blood. 2023 03 09; 141(10):1098-1099. Ayoub PG, Kohn DB. PMID: 36893006.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    24. Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials. Viruses. 2023 03 09; 15(3). Segura EER, Ayoub PG, Hart KL, Kohn DB. PMID: 36992422; PMCID: PMC10054523.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    25. Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression. Blood. 2023 03 02; 141(9):1007-1022. Wong RL, Sackey S, Brown D, Senadheera S, Masiuk K, Quintos JP, Colindres N, Riggan L, Morgan RA, Malech HL, Hollis RP, Kohn DB. PMID: 36332160; PMCID: PMC10163279.
      View in: PubMed   Mentions: 6     Fields:    Translation:Animals
    26. Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Hum Gene Ther. 2023 02; 34(3-4):83-89. McDonald CL, Qasba P, Anderson DG, Bao G, Colvin RA, Kohn DB, Malik P, Mitchell MJ, Pu WT, Rawlings DJ, Williams DA, Flotte TR. PMID: 36794978; PMCID: PMC10081695.
      View in: PubMed   Mentions:    Fields:    
    27. Updated Management Guidelines for Adenosine Deaminase Deficiency. J Allergy Clin Immunol Pract. 2023 06; 11(6):1665-1675. Grunebaum E, Booth C, Cuvelier GDE, Loves R, Aiuti A, Kohn DB. PMID: 36736952.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    28. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. J Clin Oncol. 2023 04 20; 41(12):2227-2237. Eapen M, Brazauskas R, Williams DA, Walters MC, St Martin A, Jacobs BL, Antin JH, Bona K, Chaudhury S, Coleman-Cowger VH, DiFronzo NL, Esrick EB, Field JJ, Fitzhugh CD, Kanter J, Kapoor N, Kohn DB, Krishnamurti L, London WB, Pulsipher MA, Talib S, Thompson AA, Waller EK, Wun T, Horowitz MM. PMID: 36623245; PMCID: PMC10448940.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    29. Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials. Mol Ther. 2023 03 01; 31(3):801-809. Cornetta K, Yao J, House K, Duffy L, Adusumilli PS, Beyer R, Booth C, Brenner M, Curran K, Grilley B, Heslop H, Hinrichs CS, Kaplan RN, Kiem HP, Kochenderfer J, Kohn DB, Mailankody S, Norberg SM, O'Cearbhaill RE, Pappas J, Park J, Ramos C, Ribas A, Rivière I, Rosenberg SA, Sauter C, Shah NN, Slovin SF, Thrasher A, Williams DA, Lin TY. PMID: 36518078; PMCID: PMC10014217.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCells
    30. Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing. Mol Ther Methods Clin Dev. 2023 Mar 09; 28:28-39. Cornetta K, Lin TY, Pellin D, Kohn DB. PMID: 36588821; PMCID: PMC9791246.
      View in: PubMed   Mentions: 10  
    31. Improving Lentiviral Transduction of Human Adipose-Derived Mesenchymal Stem Cells. Hum Gene Ther. 2022 12; 33(23-24):1260-1268. Collon K, Gallo MC, Bell JA, Chang SW, Rodman JCS, Sugiyama O, Kohn DB, Lieberman JR. PMID: 35859364; PMCID: PMC9808795.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    32. The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions. J Allergy Clin Immunol. 2023 02; 151(2):539-546. Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai SY, Griffith LM, Cuvelier GDE, Eissa H, Shah AJ, O'Reilly RJ, Pulsipher MA, Wright NAM, Abraham RS, Satter LF, Notarangelo LD, Puck JM. PMID: 36456361; PMCID: PMC9905311.
      View in: PubMed   Mentions: 29     Fields:    Translation:HumansCells
    33. The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions. J Allergy Clin Immunol. 2023 02; 151(2):547-555.e5. Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai SY, Kapoor N, Satter LF, Buckley RH, O'Reilly RJ, Chandra S, Bednarski JJ, Williams O, Rayes A, Moore TB, Ebens CL, Davila Saldana BJ, Petrovic A, Chellapandian D, Cuvelier GDE, Vander Lugt MT, Caywood EH, Chandrakasan S, Eissa H, Goldman FD, Shereck E, Aquino VM, Desantes KB, Madden LM, Miller HK, Yu L, Broglie L, Gillio A, Shah AJ, Knutsen AP, Andolina JP, Joshi AY, Szabolcs P, Kapadia M, Martinez CA, Parrot RE, Sullivan KE, Prockop SE, Abraham RS, Thakar MS, Leiding JW, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Puck JM. PMID: 36456360; PMCID: PMC9905305.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    34. Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants. Blood Adv. 2022 11 08; 6(21):5732-5736. White SL, Lee TD, Toy T, Carroll JE, Polsky L, Campo Fernandez B, Davila A, Kohn DB, Chang VY. PMID: 35914227; PMCID: PMC9619180.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCTClinical Trials
    35. Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential. Gene Ther. 2023 04; 30(3-4):369-376. Collon K, Bell JA, Gallo MC, Chang SW, Bougioukli S, Sugiyama O, Tassey J, Hollis R, Heckmann N, Oakes DA, Longjohn DB, Evseenko D, Kohn DB, Lieberman JR. PMID: 36216880; PMCID: PMC10086075.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimalsCells
    36. Diverse Approaches to Gene Therapy of Sickle Cell Disease. Annu Rev Med. 2023 01 27; 74:473-487. White SL, Hart K, Kohn DB. PMID: 36067800; PMCID: PMC11001465.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansCells
    37. Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC. Blood. 2022 08 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; PMCID: PMC9389638.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    38. Aberrant T-cell exhaustion in severe combined immunodeficiency survivors with poor T-cell reconstitution after transplantation. J Allergy Clin Immunol. 2023 01; 151(1):260-271. Labrosse R, Boufaied I, Bourdin B, Gona S, Randolph HE, Logan BR, Bourbonnais S, Berthe C, Chan W, Buckley RH, Parrott RE, Cuvelier GDE, Kapoor N, Chandra S, Dávila Saldaña BJ, Eissa H, Goldman FD, Heimall J, O'Reilly R, Chaudhury S, Kolb EA, Shenoy S, Griffith LM, Pulsipher M, Kohn DB, Notarangelo LD, Pai SY, Cowan MJ, Dvorak CC, Haddad É, Puck JM, Barreiro LB, Decaluwe H. PMID: 35987350; PMCID: PMC9924130.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    39. Apheresis of Deceased Donors as a New Source of Mobilized Peripheral Blood Hematopoietic Stem Cells for Transplant Tolerance. Transplantation. 2023 02 01; 107(2):504-510. Sosa RA, Mone T, Naini BV, Kohn DB, Reed EF, Wheeler K, Campo-Fernandez B, Davila A, Chaffin DJ, DiNorcia J, Kaldas FM, Cohen A, Lum EL, Veale JL, Kogut NM. PMID: 35974436; PMCID: PMC9877104.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
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    53. 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.
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    63. 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.
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    192. 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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    193. 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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    194. 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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    195. 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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    196. 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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    197. 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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    198. 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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    199. 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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    200. 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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    201. 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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    202. 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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    203. 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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    204. 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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    205. 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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    206. Lentiviral vectors ready for prime-time. Nat Biotechnol. 2007 Jan; 25(1):65-6. Kohn DB. PMID: 17211402.
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    207. 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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    208. 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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    209. 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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    210. 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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    211. 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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    212. 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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    213. 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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    214. 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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    215. 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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    216. 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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    217. 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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    218. 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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    219. 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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    220. 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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    221. 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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    222. 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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    223. 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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    224. 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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    225. 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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    226. 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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    227. 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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    228. 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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    229. 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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    230. 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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    231. 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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    232. 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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    233. 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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    234. 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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    235. 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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    236. 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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    237. 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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    238. 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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    239. 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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    240. 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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    241. 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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    242. 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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    243. 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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    244. 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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    245. 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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    246. 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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    247. [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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    248. 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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    249. 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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    250. 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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    251. 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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    252. 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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    253. 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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    254. Adenosine deaminase gene therapy protocol revisited. Mol Ther. 2002 Feb; 5(2):96-7. Kohn DB. PMID: 11829515.
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    255. 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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    256. 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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