Gori, J.L.; Haddad, E.; Frangoul, H.; Kohn, D.B.; Morris, E.C.; Martin, B.N.; Deary, B.A.; Nickerson, M.; Scholz, R.L.; Fernandez, I.; et al. 基因组编辑技术用于p47phox缺陷型慢性肉芽肿病。N. Engl. J. Med. 2025. [Google Scholar] [CrossRef] [PubMed]

NEW ENGLAND JOURNAL OF MEDICINE

IF78.5

2025-12-010

PRE

Gori, Jennifer L.; Haddad, Elie; Frangoul, Haydar; Kohn, Donald B.; Morris, Emma C.; Martin, Bradley N.; Deary, Briana A.; Nickerson, McKinley; Scholz, Rebecca L.; Fernandez, Isabel; Leveille, Karine; De Ravin, Suk See; Kang, Elizabeth M.; Pierzynski, Marie; Estwick, Tyra; Littel, Patricia; Kuhns, Douglas B.; Long Priel, Debra A.; Teira, Pierre; Turvey, Stuart; Arnold, Jordyn; Evans, Misty D.; McManus, Meghann; Carpenter, Ben; Waterman, David P.; Anzalone, Andrew V.; Petrusich, Alexandria; Osuna, Christa E.; O'Malley, Tiernan T.; Stewart-Ornstein, Jacob; Heath, Jack M.; Nehilla, Barrett J.; Asmal, Mohammed; Malech, Harry L.

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency

腺苷脱氨酶缺乏症的基因治疗长期安全性与有效性

NEW ENGLAND JOURNAL OF MEDICINE

IF78.5

2025-10-160

PRE

Booth, Claire; Masiuk, Katelyn; Vazouras, Konstantinos; Fernandes, Augustine; Xu-Bayford, Jinhua; Campo Fernandez, Beatriz; Roy, Sohini; Curio-Penny, Beatrice; Arnold, Jordyn; Terrazas, Dayna; Reid, Jack; Gilmour, Kimberly C.; Adams, Stuart; Alvarez Mediavilla, Elena; Mhaldien, Lana; O'Toole, Grainne; Ahmed, Rima; Garabedian, Elizabeth; Malech, Harry; De Ravin, Suk See; Moore, Theodore B.; De Oliveira, Satiro; Pellin, Danilo; Lin, Tsai-Yu; Dang, Thao Thi; Cornetta, Kenneth; Hershfield, Michael S.; Hara, Havinder; Thrasher, Adrian J.; Gaspar, H. Bobby; Kohn, Donald B.

Umbilical Cord Blood Transplantation Provides an Alternative for Patients with Chronic Granulomatous Disease Lacking HLA-Matched Donors: a PIDTC Report

脐带血移植为缺乏HLA匹配供体的慢性肉芽肿病患者提供了替代方案：一份PIDTC报告

Transplantation and Cellular Therapy

IF4.4

2025-10-020

PRE

Danielle E. Arnold; Jennifer W. Leiding; Brent Logan; Rebecca A. Marsh; Linda M. Griffith; Eyal Grunebaum; Luis Murguía-Favela; Kanwaldeep Mallhi; Deepakbabu Chellapandian; Christin L. Deal; Stephanie Si Lim; Vinod Prasad; Jennifer Heimall; Shanmuganathan Chandrakasan; Karin Chen; Lolie C. Yu; Christine M. Seroogy; Alfred Gillio; Jeffrey J. Bednarski; Neena Kapoor; Theodore B. Moore; Geoffrey D.E. Cuvelier; Fabien Touzot; Ahmad Rayes; Christen L. Ebens; Edo Schaefer; Andrea Bauchat; Ashley Chopek; Lauri Burroughs; Morton J. Cowan; Christopher C. Dvorak; Elie Haddad; Donald B. Kohn; Luigi D. Notarangelo; Sung-Yun Pai; Jennifer M. Puck; Michael A. Pulsipher; Troy Torgerson; Harry L. Malech; Elizabeth M. Kang; Suhag Parikh

Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells

慢病毒载体用于造血干细胞基因治疗，可恢复α-地中海贫血红细胞中的α-球蛋白表达

Cell Reports Medicine

IF10.6

2025-09-170

OAAI

Hematopoietic Stem Cell Gene Therapy for the Treatment of X-Linked Agammaglobulinemia

造血干细胞基因治疗在X连锁无丙种球蛋白血症治疗中的应用

Molecular Therapy Methods & Clinical Development

IF4.7

2025-08-120

OAAI

Quantifying the mutational landscape of retroviral and lentiviral vectors in gene therapy patients

量化基因治疗患者中反转录病毒和慢病毒载体的突变景观

Molecular Therapy

IF12

2025-05-300

OAAI

Making sense of adenosine deaminase variants and their clinical implications

阐明腺苷脱氨酶变体及其临床意义

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

IF11.2

2025-01-010

PRE

Enhancing pediatric access to cell and gene therapies

提高儿科获得细胞和基因疗法的机会

NATURE MEDICINE

IF50

2024-06-172

PRE

Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method

NATURE BIOTECHNOLOGY

IF41.7

2024-05-149

OAAI

Li, Yan-Ruide; Zhou, Yang; Yu, Jiaji; Kim, Yu Jeong; Li, Miao; Lee, Derek; Zhou, Kuangyi; Chen, Yuning; Zhu, Yichen; Wang, Yu-Chen; Li, Zhe; Yu, Yanqi; Dunn, Zachary Spencer; Guo, Wenbin; Cen, Xinjian; Husman, Tiffany; Bajpai, Aarushi; Kramer, Adam; Wilson, Matthew; Fang, Ying; Huang, Jie; Li, Shuo; Zhou, Yonggang; Zhang, Yuchong; Hahn, Zoe; Zhu, Enbo; Ma, Feiyang; Pan, Calvin; Lusis, Aldons J.; Zhou, Jin J.; Seet, Christopher S.; Kohn, Donald B.; Wang, Pin; Zhou, Xianghong Jasmine; Pellegrini, Matteo; Puliafito, Benjamin R.; Larson, Sarah M.; Yang, Lili

Donald B. Kohn

个人简介

教育背景

工作经历

荣誉奖项

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