Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy

被引：0

作者：

Walters, Mark C. ^{[1
]}

Locatelli, Franco ^{[2
]}

Thrasher, Adrian J. ^{[3
]}

Tisdale, John F. ^{[4
]}

Orchard, Paul J. ^{[5
]}

Duncan, Christine N. ^{[6
,7
]}

Kuehl, Joern-Sven ^{[8
]}

De Oliveira, Satiro Nakamura ^{[9
]}

Sauer, Martin G. ^{[10
]}

Kulozik, Andreas E. ^{[11
]}

Yannaki, Evangelia ^{[12
]}

Hongeng, Suradej ^{[13
]}

Mapara, Markus Y. ^{[14
]}

Krishnamurti, Lakshmanan ^{[15
]}

Hermine, Olivier ^{[16
]}

Blanche, Stephane ^{[17
,18
]}

Aubourg, Patrick ^{[19
,20
]}

Smith, Nicholas J. C. ^{[21
]}

Shi, Weiliang ^{[22
]}

Colvin, Richard A. ^{[22
]}

McNeil, Elizabeth ^{[22
]}

Ribeil, Jean-Antoine ^{[22
]}

Cavazzana, Marina ^{[23
,24
]}

Williams, David A. ^{[7
,25
,26
,27
]}

机构：

[1] UCSF Benioff Childrens Hosp, Oakland, CA USA

[2] IRCCS Bambino Gesu Childrens Hosp, Dept Hematol Oncol Cell & Gene Therapy, Rome, Italy

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

[4] NHLBI, Sickle Cell Branch, NIDDK, NIH, Bldg 10, Bethesda, MD 20892 USA

[5] Univ Minnesota, Mason Childrens Hosp, Minneapolis, MN USA

[6] Dana Farber Canc Inst, Pediat Oncol, Boston, MA 02115 USA

[7] Boston Childrens Hosp, Boston, MA USA

[8] Univ Clin Leipzig, Leipzig, Germany

[9] Univ Calif Los Angeles, Los Angeles, CA USA

[10] Hannover Med Sch, Pediat Hematol & Oncol, Hannover, Germany

[11] Heidelberg Univ, EMBL, MMPU, Heidelberg, Germany

[12] George Papanikolaou Hosp, Hematol HCT Unit, Gene & Cell Therapy Ctr, Thessaloniki, Greece

[13] Mahidol Univ, Ramathibodi Hosp, Bangkok, Thailand

[14] Columbia Univ, Med Ctr, Div Hematol Oncol, Columbia Ctr Translat Immunol, New York, NY USA

[15] Emory Univ, Sch Med, Aflac Canc & Blood Disorders Ctr, Childrens Healthcare Atlanta, Atlanta, GA USA

[16] Hop Necker Enfants Malad, Adult Hematol, Paris, France

[17] Hop Necker Enfants Malad, Pediat Hematol Immunol Rhumatol Unit, Paris, France

[18] Univ Paris 05, Sorbonne Paris Cite, Paris, France

[19] INSERM, Paris, France

[20] Hop Bicetre, Paris, France

[21] Womens & Childrens Hosp, Adelaide, SA, Australia

[22] Bluebird Bio Inc, Cambridge, MA USA

[23] Univ Paris 05, Sorbonne Paris Cite, IMAGINE Inst, Paris, France

[24] Hop Necker Enfants Malad, Biotherapy, Paris, France

[25] Harvard Med Sch, Boston, MA 02115 USA

[26] Harvard Stem Cell Inst, Boston, MA USA

[27] Dana Farber Boston Childrens Canc & Blood Disorde, Boston, MA USA

来源：

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION | 2020年 / 26卷 / 03期

关键词：

D O I：

暂无

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：S38 / S39

页数：2