A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
About this item
Full title
Author / Creator
Hacein-Bey-Abina, Salima , Pai, Sung-Yun , Gaspar, H. Bobby , Armant, Myriam , Berry, Charles C , Blanche, Stephane , Bleesing, Jack , Blondeau, Johanna , de Boer, Helen , Buckland, Karen F , Caccavelli, Laure , Cros, Guilhem , De Oliveira, Satiro , Fernández, Karen S , Guo, Dongjing , Harris, Chad E , Hopkins, Gregory , Lehmann, Leslie E , Lim, Annick , London, Wendy B , van der Loo, Johannes C.M , Malani, Nirav , Male, Frances , Malik, Punam , Marinovic, M. Angélica , McNicol, Anne-Marie , Moshous, Despina , Neven, Benedicte , Oleastro, Matías , Picard, Capucine , Ritz, Jerome , Rivat, Christine , Schambach, Axel , Shaw, Kit L , Sherman, Eric A , Silberstein, Leslie E , Six, Emmanuelle , Touzot, Fabien , Tsytsykova, Alla , Xu-Bayford, Jinhua , Baum, Christopher , Bushman, Frederic D , Fischer, Alain , Kohn, Donald B , Filipovich, Alexandra H , Notarangelo, Luigi D , Cavazzana, Marina , Williams, David A and Thrasher, Adrian J
Publisher
Waltham, MA: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
Waltham, MA: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Correction of the genetic lesion leading to X-linked SCID with first-generation retroviral vectors has been associated with a 25% risk of acute leukemia. A self-inactivating retrovirus appears to retain therapeutic efficacy, with no leukemia yet observed.
X-linked severe combined immunodeficiency (SCID-X1) is caused by mutations in the gene enco...
Alternative Titles
Full title
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
Authors, Artists and Contributors
Author / Creator
Pai, Sung-Yun
Gaspar, H. Bobby
Armant, Myriam
Berry, Charles C
Blanche, Stephane
Bleesing, Jack
Blondeau, Johanna
de Boer, Helen
Buckland, Karen F
Caccavelli, Laure
Cros, Guilhem
De Oliveira, Satiro
Fernández, Karen S
Guo, Dongjing
Harris, Chad E
Hopkins, Gregory
Lehmann, Leslie E
Lim, Annick
London, Wendy B
van der Loo, Johannes C.M
Malani, Nirav
Male, Frances
Malik, Punam
Marinovic, M. Angélica
McNicol, Anne-Marie
Moshous, Despina
Neven, Benedicte
Oleastro, Matías
Picard, Capucine
Ritz, Jerome
Rivat, Christine
Schambach, Axel
Shaw, Kit L
Sherman, Eric A
Silberstein, Leslie E
Six, Emmanuelle
Touzot, Fabien
Tsytsykova, Alla
Xu-Bayford, Jinhua
Baum, Christopher
Bushman, Frederic D
Fischer, Alain
Kohn, Donald B
Filipovich, Alexandra H
Notarangelo, Luigi D
Cavazzana, Marina
Williams, David A
Thrasher, Adrian J
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4274995
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4274995
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1404588
