Development of Gene Therapy for Two Blood DisordersWednesday, 20 July 2011 at 09:30 Add to Calendar ▼SELECTBIOenquiries@selectbiosciences.com Clinical gene therapy for inherited immunodeficiencies has been successful in the clinic since approximately 50 patients with these lethal disorders have been treated successfully. To develop gene therapy of hematopoietic stem cells for serious disorders that are not life-threatening, safer vectors and milder bone marrow ablation techniques will need to be developed. This presentation will address how we can use mouse models for Diamond Blackfan anemia and Gaucher disease to develop safe gene therapy for these disorders. |