Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy

被引：13

作者：

Magrin, Elisa ^{[1
,2
]}

Semeraro, Michaela ^{[1
]}

Magnani, Alessandra ^{[3
,4
]}

Puy, Herve ^{[5
]}

Miccio, Annarita ^{[6
]}

Hebert, Nicolas ^{[7
,8
,9
]}

Diana, Jean-Sebastien

Lefrere, Francois

Suarez, Felipe

Hermine, Olivier

Brousse, Valentine

Poirot, Catherine

Bourget, Philippe

El Nemer, Wassim

Guichard, Isabelle

Moshous, Despina

Neven, Benedicte ^{[6
,10
]}

Monpoux, Fabrice ^{[11
]}

Poiree, Marilyne ^{[11
]}

Bartolucci, Pablo ^{[9
,12
]}

Meritet, Jean-Francois ^{[13
]}

Grevent, David ^{[14
]}

Lefebvre, Thibaud ^{[15
]}

Asmal, Mohammed ^{[16
]}

Whitney, Erin ^{[16
]}

Gayron, Marisa ^{[16
]}

Huang, Wenmei ^{[16
]}

Funck-Brentano, Isabelle ^{[1
]}

de Montalembert, Mariane ^{[17
]}

Joseph, Laure ^{[18
]}

Ribeil, Jean-Antoine ^{[16
]}

Cavazzana, Marina ^{[19
]}

机构：

[1] Necker Childrens Hosp, Assistance Publ Hop Paris, Paris, France

[2] Necker Assistance Publ Hop Paris, Biotherapy Dept, Paris, France

[3] Necker Enfants Malad Univ Hosp, Paris, France

[4] Grp Hosp Univ Ouest, Assistance Publ Hop Paris, Biotherapy Clin Invest Ctr, Paris, France

[5] Univ Paris Diderot, Sorbonne Paris Cite, Paris, France

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

[7] Univ Paris Est Creteil, Creteil, France

[8] Hop Henri Mondor, Etab Francais Sang, Creteil, France

[9] Univ Paris Est Creteil, Inst Mondor, Lab Excellence GR Ex, INSERM U955,EFS,Team Transfus & Malad Globule Rou, Creteil, France

[10] Hop Necker Enfants Malad, Paris, France

[11] Univ Hosp Nice, Nice, France

[12] Henri Mondor Hosp, APHP, Creteil, France

[13] Hop Cochin St Vincent Paul, Paris, France

[14] Necker Childrens Hosp, Dept Pediat Neuroradiol, Paris, France

[15] Univ Paris, Louis Mourier Hosp, Assistance Publ Hop Paris, Paris, France

[16] Bluebird Bio Inc, Cambridge, MA USA

[17] Hop Necker Enfants Malad, APHP, Referral Ctr Sickle Cell Dis, Pediat, Paris, France

[18] Necker Childrens Hosp, Assistance Publ Hop Paris, Biotherapy Dept, Paris, France

[19] Hop Necker Enfants Malad, Grp Hosp Univ Ouest, Biotherapy Clin Invest Ctr, Paris, France

来源：

BLOOD | 2019年 / 134卷

关键词：

D O I：

10.1182/blood-2019-127393

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页数：4

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