Gene Therapy for Hemophilia: Past, Present and Future

After numerous pre-clinical studies demonstrated consistent success in large and small animal models, gene therapy has finally seen initial signs of clinically meaningful success. In a landmark study, Nathwani and colleagues reported sustained factor IX (FIX) expression in individuals with severe hemophilia B following adeno-associated virus (AAV)-mediated in vivo factor IX (FIX) gene transfer. As the next possible treatment-changing paradigm in hemophilia care, gene therapy may provide patients with sufficient hemostatic improvement to achieve the World Federation of Hemophilia’s aspirational goal of “integration of opportunities in all aspects of life… equivalent to someone without a bleeding disorder.” Although promising momentum supports the potential of gene therapy to replace protein-based therapeutics for hemophilia, several obstacles remain.
Source: Seminars in Hematology - Category: Hematology Authors: Source Type: research