Gene Therapy for Hemophilia: Innovations, Milestones, and Future Prospects

Area of Science:

• Medical Genetics
• Hematology
• Biotechnology

Background:

• Haemophilia is a genetic bleeding disorder caused by deficiencies in clotting factors VIII or IX.
• Conventional treatments involve frequent intravenous factor infusions, facing challenges in cost, access, and adherence.
• Gene therapy presents a potential curative approach by enabling endogenous clotting factor production.

Purpose of the Study:

• To highlight recent breakthroughs in gene therapy for haemophilia, particularly contributions from Indian scientists.
• To discuss the application of adeno-associated viral vectors and genome editing technologies.
• To explore the potential of gene therapy to transition haemophilia management towards curative strategies.

Main Methods:

• Utilizing adeno-associated viral vectors for efficient and safe gene delivery.
• Integrating advanced genome editing technologies like CRISPR/Cas9.
• Reviewing clinical advancements and research in haemophilia gene therapy.

Main Results:

• Gene therapy demonstrates potential to overcome limitations of conventional treatments, such as inhibitor development and repetitive infusions.
• Indian scientists have made significant contributions to pioneering gene therapy approaches for haemophilia.
• The technology promises long-term efficacy and improved quality of life for patients.

Conclusions:

• Gene therapy, especially with viral vectors and genome editing, offers a transformative shift from routine haemophilia care to potential cures.
• Addressing challenges like affordability and equitable access is crucial for widespread implementation.
• This field holds significant promise for redefining therapeutic possibilities for chronic bleeding disorders.