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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
10.1056/NEJMoa2027675
000649467400001
33974366
Kohn
D.B.
author
Booth
C.
author
Shaw
K.L.
author
Xu-Bayford
J.
author
Garabedian
E.
author
Trevisan
V.
author
Carbonaro-Sarracino
D.A.
author
Soni
K.
author
Terrazas
D.
author
Snell
K.
author
Ikeda
A.
author
Leon-Rico
D.
author
Moore
T.B.
author
Buckland
K.F.
author
Shah
A.J.
author
Gilmour
K.C.
author
De Oliveira
S.
author
Rivat
C.
author
Crooks
G.M.
author
Izotova
N.
author
Tse
J.
author
Adams
S.
author
Shupien
S.
author
Ricketts
H.
author
Davila
A.
author
Uzowuru
C.
author
Icreverzi
A.
author
Barman
P.
author
Campo Fernandez
B.
author
Hollis
R.P.
author
Coronel
M.
author
Yu
A.
author
Chun
K.M.
author
Casas
C.E.
author
Zhang
R.
author
Arduini
S.
author
Lynn
F.
author
Kudari
M.
author
Spezzi
A.
author
Zahn
M.
author
Heimke
R.
author
Labik
I.
author
Parrott
R.
author
Buckley
R.H.
author
Reeves
L.
author
Cornetta
K.
author
Sokolic
R.
author
Hershfield
M.
author
Schmidt
M.
author
Candotti
F.
author
Malech
H.L.
author
Thrasher
A.J.
author
Gaspar
H.B.
author
article
2021-05-27
The New England journal of medicine
1533-4406
0028-4793
journal
384
21
2002-2013
Adenosine Deaminase/deficiency
Adolescent
Agammaglobulinemia/therapy
Child
Child, Preschool
Genetic Therapy/adverse effects
Genetic Therapy/methods
Genetic Vectors
Hematopoietic Stem Cell Transplantation
Humans
Infant
Lentivirus/genetics
Lymphocyte Count
Progression-Free Survival
Prospective Studies
Severe Combined Immunodeficiency/therapy
Transplantation, Autologous
eng
60_published
peer-reviewed
Publication types: Clinical Trial, Phase I ; Clinical Trial, Phase II ; Journal Article ; Multicenter Study ; Research Support, N.I.H., Extramural ; Research Support, N.I.H., Intramural ; Research Support, Non-U.S. Gov't
Publication Status: ppublish