Stem Cell Gene Therapy for Blood Cell Diseases

Conférence scientifique | Centre de recherche du CHU Sainte-Justine

Conférencier:

Donald B. Kohn, MD, professeur au Départements de microbiologie, immunologie et génétique moléculaire; et pédiatrie, Université de Californie à Los Angeles (U.C.L.A.)

Résumé:
The genetic blood cell disorders, such as hemoglobinopathies, primary immune deficiency diseases and lysosomal storage diseases may be treated by transplantation of autologous hematopoietic stem cells after the ex vivo gene correction. The approaches being used and current results and future challenges will be presented.