Stem Cell Gene Therapy of Hemophilia A
In the Tuesday morning session on gene therapy, Christopher B. Doering presents advances in stem cell gene therapy of hemophilia A.
Hemophilia A Gene Therapy: AAV-mediated delivery of an enhanced F8 cassette to the liver produces supraphysiological levels of human FVIII in vivo
In the Tuesday morning session on gene therapy, Brigit E. Riley explains her approach to the AAV-mediated delivery of an enhanced F8 cDNA.