Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
About this item
Full title
Author / Creator
Eichler, Florian , Duncan, Christine , Musolino, Patricia L , Orchard, Paul J , De Oliveira, Satiro , Thrasher, Adrian J , Armant, Myriam , Dansereau, Colleen , Lund, Troy C , Miller, Weston P , Raymond, Gerald V , Sankar, Raman , Shah, Ami J , Sevin, Caroline , Gaspar, H. Bobby , Gissen, Paul , Amartino, Hernan , Bratkovic, Drago , Smith, Nicholas J.C , Paker, Asif M , Shamir, Esther , O’Meara, Tara , Davidson, David , Aubourg, Patrick and Williams, David A
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Among boys with cerebral adrenoleukodystrophy, matched donors for allogeneic transplantation are available to only a few. Infusion of CD34+ autologous stem cells transduced with a lentiviral vector prevented neurologic deterioration in 15 of 17 boys with the disease.
Alternative Titles
Full title
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
Authors, Artists and Contributors
Author / Creator
Duncan, Christine
Musolino, Patricia L
Orchard, Paul J
De Oliveira, Satiro
Thrasher, Adrian J
Armant, Myriam
Dansereau, Colleen
Lund, Troy C
Miller, Weston P
Raymond, Gerald V
Sankar, Raman
Shah, Ami J
Sevin, Caroline
Gaspar, H. Bobby
Gissen, Paul
Amartino, Hernan
Bratkovic, Drago
Smith, Nicholas J.C
Paker, Asif M
Shamir, Esther
O’Meara, Tara
Davidson, David
Aubourg, Patrick
Williams, David A
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5708849
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_5708849
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa1700554
