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. Relevance of lymphocyte proliferation to PHA in severe combined immunodeficiency (SCID) and T cell lymphopenia. Clin Immunol. 2024 Feb 15; 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.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCellsPHPublic Health
    2. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study. J Allergy Clin Immunol. 2024 Jan 28. 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.
      View in: PubMed   Mentions:    Fields:    
    3. What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity. J Allergy Clin Immunol Pract. 2024 Jan 19. Mudde ACA, Kuo CY, Kohn DB, Booth C. PMID: 38246560.
      View in: PubMed   Mentions:    Fields:    
    4. 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: 3     Fields:    Translation:Humans
    5. 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
    6. Successes and challenges in clinical gene therapy. Gene Ther. 2023 Nov; 30(10-11):738-746. Kohn DB, Chen YY, Spencer MJ. PMID: 37935854; PMCID: PMC10678346.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansCells
    7. 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
    8. 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.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    9. 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.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    10. 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:    Fields:    Translation:HumansCells
    11. Biodistribution of lentiviral transduced adipose-derived stem cells for "ex-vivo" regional gene therapy for bone repair. Gene Ther. 2023 Dec; 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
    12. 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: 5     Fields:    Translation:HumansPHPublic Health
    13. 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.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansAnimalsCells
    14. 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
    15. 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: 1     Fields:    Translation:Humans
    16. 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: 4     Fields:    Translation:Animals
    17. 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:    
    18. 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: 3     Fields:    Translation:Humans
    19. 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: 3     Fields:    Translation:Humans
    20. 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: 2     Fields:    Translation:HumansCells
    21. 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: 5  
    22. 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
    23. 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: 6     Fields:    Translation:Humans
    24. 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: 17     Fields:    Translation:HumansCells
    25. 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: 1     Fields:    Translation:HumansCTClinical Trials
    26. 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: 3     Fields:    Translation:HumansAnimalsCells
    27. 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.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCells
    28. 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: 13     Fields:    Translation:HumansCTClinical Trials
    29. 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
    30. 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
    31. Findings on Optical Coherence Tomography in Malignant Infantile Osteopetrosis. Ophthalmic Surg Lasers Imaging Retina. 2022 07; 53(7):398-402. Campagna GA, Chew L, Pettenkofer M, Nicoletti E, Schwartz JD, Choi G, Fernandes AO, Terrazas D, Kohn DB, Tsui I. PMID: 35858232.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    32. High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation. iScience. 2022 Jun 17; 25(6):104374. Magis W, DeWitt MA, Wyman SK, Vu JT, Heo SJ, Shao SJ, Hennig F, Romero ZG, Campo-Fernandez B, Said S, McNeill MS, Rettig GR, Sun Y, Wang Y, Behlke MA, Kohn DB, Boffelli D, Walters MC, Corn JE, Martin DIK. PMID: 35633935; PMCID: PMC9130532.
      View in: PubMed   Mentions: 19  
    33. 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: 1     Fields:    Translation:HumansCells
    34. 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: 10     Fields:    Translation:HumansCellsPHPublic Health
    35. 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: 5  
    36. 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: 33     Fields:    Translation:HumansAnimalsCells
    37. 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: 15     Fields:    Translation:HumansCTClinical Trials
    38. 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: 5     Fields:    Translation:HumansAnimals
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    41. 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.
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    42. 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.
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    44. 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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    45. Gene Therapies for Primary Immune Deficiencies. Front Immunol. 2021; 12:648951. Kohn LA, Kohn DB. PMID: 33717203; PMCID: PMC7946985.
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    46. 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.
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    49. 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.
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    53. 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.
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    54. 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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    55. 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.
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    56. 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.
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    196. 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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    198. 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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    199. Lentiviral vectors ready for prime-time. Nat Biotechnol. 2007 Jan; 25(1):65-6. Kohn DB. PMID: 17211402.
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    201. 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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    202. 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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    203. 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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    205. 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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    206. 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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    207. 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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    208. 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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    209. 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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    210. 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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    212. 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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    213. 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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    214. 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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    215. 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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    216. 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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    217. 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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    218. 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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    219. 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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    220. 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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    221. 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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    222. 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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    223. 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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    224. 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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    225. 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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    226. 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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    227. 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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    229. 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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    230. 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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    231. 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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    232. 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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    233. 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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    234. 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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    235. 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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    236. 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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    237. [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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    238. 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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    239. 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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    240. 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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    241. 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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