Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
About this item
Full title
Author / Creator
Kanter, Julie , Walters, Mark C , Krishnamurti, Lakshmanan , Mapara, Markus Y , Kwiatkowski, Janet L , Rifkin-Zenenberg, Stacey , Aygun, Banu , Kasow, Kimberly A , Pierciey, Francis J , Bonner, Melissa , Miller, Alex , Zhang, Xinyan , Lynch, Jessie , Kim, Dennis , Ribeil, Jean-Antoine , Asmal, Mohammed , Goyal, Sunita , Thompson, Alexis A and Tisdale, John F
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
An unprespecified interim analysis of results from a phase 1–2 study of gene therapy for sickle cell disease shows resolution of severe vaso-occlusive events in 25 patients who could be evaluated. In the 24 months before enrollment, these patients had a median of 3.5 severe vaso-occlusive events per year.
Alternative Titles
Full title
Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease
Authors, Artists and Contributors
Author / Creator
Walters, Mark C
Krishnamurti, Lakshmanan
Mapara, Markus Y
Kwiatkowski, Janet L
Rifkin-Zenenberg, Stacey
Aygun, Banu
Kasow, Kimberly A
Pierciey, Francis J
Bonner, Melissa
Miller, Alex
Zhang, Xinyan
Lynch, Jessie
Kim, Dennis
Ribeil, Jean-Antoine
Asmal, Mohammed
Goyal, Sunita
Thompson, Alexis A
Tisdale, John F
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2609457010
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2609457010
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2117175
