Genetic Therapies for Sickle Cell Disease.
Sign Up to like & getrecommendations! Published in 2018 at "Pediatric clinics of North America"
DOI: 10.1016/j.pcl.2018.01.008
Abstract: Sickle cell disease is the most prevalent monogenic disorder worldwide and curative therapies are limited to hematopoietic stem cell transplant to the few with matched donors. Gene therapy has curative potential, whereby autologous hematopoietic stem… read more here.
Keywords: genetic therapies; cell disease; cell; therapies sickle ... See more keywords
Genetic therapies for sickle cell disease.
Sign Up to like & getrecommendations! Published in 2018 at "Seminars in hematology"
DOI: 10.1053/j.seminhematol.2018.04.014
Abstract: After decades with few novel therapeutic options for sickle cell disease (SCD), autologous hematopoietic stem cell (HSC) based genetic therapies including lentiviral gene therapy (GT), and genome editing (GE) now appear imminent. Lentiviral GT has… read more here.
Keywords: genetic therapies; cell disease; cell; therapies sickle ... See more keywords