ASH 2018 | Genetic targeting in sickle cell disease

Erica Esrick

Erica Esrick, MD, of Harvard Medical School, Boston, MA, discusses the inital results of the genetic targeting of BCL11A in sickle cell disease using a shRNAmiR lentiviral vector. This gene therapy showed promising results, as presented here at the American Society of Hematology (ASH) 2018 Annual Meeting and Exposition in San Diego, CA.

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