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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 ; Corbacioglu, Selim

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

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 (2021) CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia. New England Journal of Medicine 384 (3), pp. 252-260.

Date of publication of this fulltext: 29 Feb 2024 12:27
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Item typeArticle
Journal or Publication TitleNew England Journal of Medicine
Publisher:MASSACHUSETTS MEDICAL SOC
Place of Publication:WALTHAM
Volume:384
Number of Issue or Book Chapter:3
Page Range:pp. 252-260
Date2021
InstitutionsMedicine > Lehrstuhl für Kinder- und Jugendmedizin
Identification Number
ValueType
10.1056/NEJMoa2031054DOI
KeywordsFETAL-HEMOGLOBIN; RISK-FACTORS; BCL11A; GENOME; TRANSPLANTATION; THERAPY; ASSOCIATION; MORBIDITY; ENHANCER; BLOOD;
Dewey Decimal Classification600 Technology > 610 Medical sciences Medicine
StatusPublished
RefereedYes, this version has been refereed
Created at the University of RegensburgYes
Item ID56388

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