Treatment by CRISPR-Cas9 Gene Editing — A Proof of Principle

  • Malech, Harry L. M.D.

  • From the Genetic Immunotherapy Section, Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD.

Disclosure forms provided by the author are available with the full text of this editorial at NEJM.org.

New England Journal of Medicine 384(3):p 286-287, January 21, 2021. | DOI: 10.1056/NEJMe2034624

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

  • Frangoul, Haydar M.D.
  • Altshuler, David M.D., Ph.D.
  • Cappellini, Domenica M. M.D.
  • Chen, Yi-Shan Ph.D.
  • Domm, Jennifer M.D.
  • Eustace, Brenda K. Ph.D.
  • Foell, Juergen M.D.
  • de la Fuente, Josu M.D., Ph.D.
  • Grupp, Stephan M.D., Ph.D.
  • Handgretinger, Rupert M.D.
  • Ho, Tony W. M.D.
  • Kattamis, Antonis M.D.
  • Kernytsky, Andrew Ph.D.
  • Lekstrom-Himes, Julie M.D.
  • Li, Amanda M. M.D.
  • Locatelli, Franco M.D.
  • Mapara, Markus Y. M.D., Ph.D.
  • de Montalembert, Mariane M.D.
  • Rondelli, Damiano M.D.
  • Sharma, Akshay M.B., B.S.
  • Sheth, Sujit M.D.
  • Soni, Sandeep M.D.
  • Steinberg, Martin H. M.D.
  • Wall, Donna M.D.
  • Yen, Angela Ph.D.
  • Corbacioglu, Selim M.D.
New England Journal of Medicine 384(3):p 252-260, January 21, 2021. | DOI: 10.1056/NEJMoa2031054
Copyright © 2021 Massachusetts Medical Society. All rights reserved.
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