Gene Therapy for Sickle Cell Anemia in India - Current Status and Challenges

Area of Science:

• Hematology
• Genetics
• Medical Research

Background:

• Sickle cell disease (SCD) is a severe monogenic disorder prevalent in India, significantly impacting patient quality of life.
• Current curative treatment, allogeneic hematopoietic stem cell transplantation, has limitations.
• Gene therapy presents a promising alternative for SCD treatment.

Purpose of the Study:

• To review gene addition and gene editing technologies for SCD treatment.
• To highlight global clinical trials for SCD gene therapies.
• To examine SCD research and challenges in India.

Main Methods:

• Review of current gene addition and gene editing technologies.
• Analysis of global clinical trial data for SCD gene therapies.
• Assessment of research initiatives and challenges in India.

Main Results:

• Gene therapy, including gene addition and editing, shows significant potential for SCD cure.
• Several gene therapy interventions are in various stages of global clinical trials.
• India is actively researching SCD curative therapies but faces unique challenges.

Conclusions:

• Gene therapy offers a transformative approach to curing sickle cell disease.
• Global advancements are progressing, with Indian research showing promise.
• Addressing ethical, socio-economic, and regulatory barriers is crucial for implementing SCD gene therapies in India.