Pathogenic or Not? Classification of genetic variants in haemostasis
In the Wednesday morning lab sciences session on the genetic laboratory for bleeding disorders, Anne Goodeve explains the classification of genetic variants in haemostasis.
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.
The "My Life, Our Future" Hemophilia Genetic Analysis Project
In the Wednesday morning lab sciences session on the genetic laboratory for bleeding disorders, Barbara A. Konkle discusses the work and experiences, to date, of the My Life, Our Future hemophilia genetic analysis project.
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.