LENTIVIRAL-MEDIATED GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA [GROUP A]: UPDATED RESULTS FROM GLOBAL RP-L102 CLINICAL TRIALS

被引：0

作者：

Sevilla, Julian ^{[1
,2
]}

Booth, Claire ^{[3
]}

Czechowicz, Agnieszka ^{[4
,5
]}

Agarwal, Rajni ^{[4
,5
]}

Zubicaray, Josune ^{[1
]}

Rio, Paula ^{[2
,6
,7
]}

Chetty, Kritika ^{[3
]}

O'Toole, Grainne ^{[3
]}

Xu-Bayford, Jinhua ^{[3
]}

Ancliff, Philip ^{[3
]}

Sebastian, Elena ^{[1
]}

Choi, Grace ^{[8
]}

Zeini, Miriam ^{[8
]}

Nicoletti, Eileen ^{[8
]}

Eide, Cindy ^{[9
]}

Wagner, John ^{[9
]}

Rao, Gayatri ^{[8
]}

Thrasher, Adrian ^{[3
]}

Schwartz, Jonathan ^{[8
]}

Roncarolo, Maria Grazia ^{[4
,5
]}

Bueren, Juan ^{[2
,6
,7
]}

机构：

[1] Hosp Infantil Univ Nino Jesus HIUNJ, Fdn Invest Biomed, Madrid, Spain

[2] Ctr Invest Biomed Red Enfermedades Raras CIBERER, Madrid, Spain

[3] UCL Great Ormond St Inst Child Hlth, London, England

[4] Stanford Univ, Sch Med, Stanford, CA USA

[5] Lucile Packard Childrens Hosp, Palo Alto, CA USA

[6] Ctr Invest Energet Medioambientales & Tecnolog CI, Madrid, Spain

[7] Inst Invest Sanitaria Fdn Jimenez Diaz IIS FJD, Madrid, Spain

[8] Rocket Pharmaceut Inc, Cranbury, NJ USA

[9] Univ Minnesota, Minneapolis, MN USA

来源：

BONE MARROW TRANSPLANTATION | 2023年 / 58卷 / SUPP1期

关键词：

D O I：

暂无

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

P182

引用

页码：276 / 277

页数：2

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