CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
About this item
Full title
Author / Creator
Frangoul, Haydar , Altshuler, David , Cappellini, M. Domenica , Chen, Yi-Shan , Domm, Jennifer , Eustace, Brenda K , Foell, Juergen , de la Fuente, Josu , Grupp, Stephan , Handgretinger, Rupert , Ho, Tony W , Kattamis, Antonis , Kernytsky, Andrew , Lekstrom-Himes, Julie , Li, Amanda M , Locatelli, Franco , Mapara, Markus Y , de Montalembert, Mariane , Rondelli, Damiano , Sharma, Akshay , Sheth, Sujit , Soni, Sandeep , Steinberg, Martin H , Wall, Donna , Yen, Angela and Corbacioglu, Selim
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
Two patients, one with transfusion-dependent β-thalassemia and the other with sickle cell disease, received autologous CD34+ cells edited with CRISPR-Cas9 targeting of
BCL11A
. Their clinical course over the following 16 to 18 months supports further experimental testing of CRISPR-Cas9 gene editing to treat these diseases.
Alternative Titles
Full title
CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Authors, Artists and Contributors
Author / Creator
Altshuler, David
Cappellini, M. Domenica
Chen, Yi-Shan
Domm, Jennifer
Eustace, Brenda K
Foell, Juergen
de la Fuente, Josu
Grupp, Stephan
Handgretinger, Rupert
Ho, Tony W
Kattamis, Antonis
Kernytsky, Andrew
Lekstrom-Himes, Julie
Li, Amanda M
Locatelli, Franco
Mapara, Markus Y
de Montalembert, Mariane
Rondelli, Damiano
Sharma, Akshay
Sheth, Sujit
Soni, Sandeep
Steinberg, Martin H
Wall, Donna
Yen, Angela
Corbacioglu, Selim
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2467843820
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2467843820
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2031054
