Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
About this item
Full title
Author / Creator
Kohn, Donald B , Booth, Claire , Shaw, Kit L , Xu-Bayford, Jinhua , Garabedian, Elizabeth , Trevisan, Valentina , Carbonaro-Sarracino, Denise A , Soni, Kajal , Terrazas, Dayna , Snell, Katie , Ikeda, Alan , Leon-Rico, Diego , Moore, Theodore B , Buckland, Karen F , Shah, Ami J , Gilmour, Kimberly C , De Oliveira, Satiro , Rivat, Christine , Crooks, Gay M , Izotova, Natalia , Tse, John , Adams, Stuart , Shupien, Sally , Ricketts, Hilory , Davila, Alejandra , Uzowuru, Chilenwa , Icreverzi, Amalia , Barman, Provaboti , Campo Fernandez, Beatriz , Hollis, Roger P , Coronel, Maritess , Yu, Allen , Chun, Krista M , Casas, Christian E , Zhang, Ruixue , Arduini, Serena , Lynn, Frances , Kudari, Mahesh , Spezzi, Andrea , Zahn, Marco , Heimke, Rene , Labik, Ivan , Parrott, Roberta , Buckley, Rebecca H , Reeves, Lilith , Cornetta, Kenneth , Sokolic, Robert , Hershfield, Michael , Schmidt, Manfred , Candotti, Fabio , Malech, Harry L , Thrasher, Adrian J and Gaspar, H. Bobby
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
Fifty patients in the United States and the United Kingdom underwent HSCT with CD34+ cells transduced with a replication-defective lentivirus bearing
ADA
. At 24 months, all the patients were alive, and event-free survival was 96.7% among the U.S. patients and 95% among the U.K. patients. Only two patients did not have persistent engraftment....
Alternative Titles
Full title
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency
Authors, Artists and Contributors
Author / Creator
Booth, Claire
Shaw, Kit L
Xu-Bayford, Jinhua
Garabedian, Elizabeth
Trevisan, Valentina
Carbonaro-Sarracino, Denise A
Soni, Kajal
Terrazas, Dayna
Snell, Katie
Ikeda, Alan
Leon-Rico, Diego
Moore, Theodore B
Buckland, Karen F
Shah, Ami J
Gilmour, Kimberly C
De Oliveira, Satiro
Rivat, Christine
Crooks, Gay M
Izotova, Natalia
Tse, John
Adams, Stuart
Shupien, Sally
Ricketts, Hilory
Davila, Alejandra
Uzowuru, Chilenwa
Icreverzi, Amalia
Barman, Provaboti
Campo Fernandez, Beatriz
Hollis, Roger P
Coronel, Maritess
Yu, Allen
Chun, Krista M
Casas, Christian E
Zhang, Ruixue
Arduini, Serena
Lynn, Frances
Kudari, Mahesh
Spezzi, Andrea
Zahn, Marco
Heimke, Rene
Labik, Ivan
Parrott, Roberta
Buckley, Rebecca H
Reeves, Lilith
Cornetta, Kenneth
Sokolic, Robert
Hershfield, Michael
Schmidt, Manfred
Candotti, Fabio
Malech, Harry L
Thrasher, Adrian J
Gaspar, H. Bobby
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8240285
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8240285
Other Identifiers
ISSN
0028-4793,1533-4406
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2027675
