Published in
American Association for the Advancement of Science, Science Translational Medicine, 598(13), 2021
DOI: 10.1126/scitranslmed.abf2444
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Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease
Journal article published in 2021 by
Annalisa Lattanzi 
,
Joab Camarena ,
Premanjali Lahiri ,
Helen Segal,
Waracharee Srifa,
Christopher A. Vakulskas,
Richard L. Frock ,
Josefin Kenrick ,
Ciaran Lee ,
Narae Talbott,
Jason Skowronski ,
M. Kyle Cromer ,
Carsten T. Charlesworth ,
Rasmus O. Bak,
Sruthi Mantri
and other authors.
,
Joab Camarena ,
Premanjali Lahiri ,
Helen Segal,
Waracharee Srifa,
Christopher A. Vakulskas,
Richard L. Frock ,
Josefin Kenrick ,
Ciaran Lee ,
Narae Talbott,
Jason Skowronski ,
M. Kyle Cromer ,
Carsten T. Charlesworth ,
Rasmus O. Bak,
Sruthi Mantri
and other authors.
This paper was not found in any repository, but could be made available legally by the author.
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Abstract
Preclinical efficacy and safety data in mice provide support for ex vivo β-globin gene correction to treat patients with sickle cell disease.