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ASH 2018 | Genetic targeting in sickle cell disease

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.