Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy.
Sadelain
Michel
M
Gene Transfer and Gene Expression Laboratory, Department of Medicine and Immunology Program, Memorial Sloan-Kettering Cancer Center, New York, NY 10021, USA. m-sadelain@ski.mskcc.org
Boulad
Farid
F
Galanello
Renzo
R
Giardina
Patricia
P
Locatelli
Franco
F
Maggio
Aurelio
A
Rivella
Stefano
S
Riviere
Isabelle
I
Tisdale
John
J
D000818:Animals; D015316:Genetic Therapy / Q000379:methods; D005914:Globins / Q000235:genetics*; D006801:Humans; D051379:Mice; D017086:beta-Thalassemia / Q000235:genetics / Q000473:pathology / Q000628:therapy*
D005914:Globins