Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy

Title:Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy

Volume: 24 Issue: 4

Author(s): Safir Ullah Khan*, Munir Ullah Khan, Muhammad Suleman, Amrah Inam and Muhammad Azhar Ud Din

Affiliation:

• Hefei National Laboratory for Physical Sciences at the Microscale, School of Life Sciences, University of Science and Technology of China, Hefei, 230027, People’s Republic of China

Abstract: Gene therapy for hemophilia has advanced tremendously after thirty years of continual study and development. Advancements in medical science have facilitated attaining normal levels of Factor VIII (FVIII) or Factor IX (FIX) in individuals with haemophilia, thereby offering the potential for their complete recovery. Despite the notable advancements in various countries, there is significant scope for further enhancement in haemophilia gene therapy. Adeno-associated virus (AAV) currently serves as the primary vehicle for gene therapy in clinical trials targeting haemophilia. Subsequent investigations will prioritize enhancing viral capsid structures, transgene compositions, and promoters to achieve heightened transduction efficacy, diminished immunogenicity, and more predictable therapeutic results. The present study indicates that whereas animal models have transduction efficiency that is over 100% high, human hepatocytes are unable to express clotting factors and transduction efficiency to comparable levels. According to the current study, achieving high transduction efficiency and high levels of clotting factor expression in human hepatocytes is still insufficient. It is also crucial to reduce the risk of cellular stress caused by protein overload. Despite encountering various hurdles, the field of haemophilia gene therapy holds promise for the future. As technology continues to advance and mature, it is anticipated that a personalized therapeutic approach will be developed to cure haemophilia effectively.

Cite this article as:

Khan Ullah Safir*, Khan Ullah Munir, Suleman Muhammad, Inam Amrah and Din Ud Muhammad Azhar, Hemophilia Healing with AAV: Navigating the Frontier of Gene Therapy, Current Gene Therapy 2024; 24 (4) . https://dx.doi.org/10.2174/0115665232279893231228065540