Clinical Edge

Summaries of Must-Read Clinical Literature, Guidelines, and FDA Actions

Must Reads In Gene Therapy

Gene Therapy for January/February 2020

Gene therapy effective in hemophilia B patients with neutralizing antibodies,

More must reads

Gene Therapy for November/December 2019

BCL11A-directed gene therapy advances in sickle cell disease,

More must reads

Gene Therapy for March/April 2019

SB-525 Gene Therapy Promising for Hemophilia A,

Padua variant factor IX gene shines in AMT-061,

More must reads

Gene Therapy for November/December 2018

Gene therapy reverses sickle cell phenotype in small study, Esrick EB et al. ASH 2018, Abstract 1023

Gene therapy in severe hemophilia B, Reiss UM et al. ASH 2018, Abstract 491
More must reads

Gene Therapy for September/October 2018

Feds move to streamline oversight of gene therapy, Collins FS and Gottlieb S. N Engl J Med. doi: 10.1056/NEJMp1810628
More must reads