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Comment
. 2020 Dec;588(7838):383.
doi: 10.1038/d41586-020-03476-x.

CRISPR gene therapy shows promise against blood diseases

Heidi Ledford
Comment

CRISPR gene therapy shows promise against blood diseases

Heidi Ledford. Nature. 2020 Dec.
No abstract available

Keywords: CRISPR-Cas9 genome editing; Gene therapy; Genetics.

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Comment on

  • CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
    Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. Frangoul H, et al. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283989 Clinical Trial.
  • Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease.
    Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, Abriss D, Boardman K, Khelladi R, Shaw K, Negre H, Negre O, Nikiforow S, Ritz J, Pai SY, London WB, Dansereau C, Heeney MM, Armant M, Manis JP, Williams DA. Esrick EB, et al. N Engl J Med. 2021 Jan 21;384(3):205-215. doi: 10.1056/NEJMoa2029392. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283990 Free PMC article. Clinical Trial.

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    1. Frangoul, H. et al. N. Engl J. Med. https://doi.org/10.1056/NEJMoa2031054 (2020). - DOI - PubMed
    1. Esrick, E. B. et al. N. Engl J. Med. https://doi.org/10.1056/NEJMoa2029392 (2020). - DOI - PubMed

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