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4:30
YouTube
ASGCT
Gene Therapy and Sickle Cell Disease
Sickle cell disease is an inherited blood disorder that causes red blood cells to deform and become sickle (or crescent) shaped. Gene therapy and gene editing may offer a one-time treatment that addresses the genetic cause of sickle cell disease. The subtitling of these videos was funded in part by a donation from Pfizer, Inc.
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