Rare genetic disorders in India: Current status, challenges, and CRISPR-based therapy

Area of Science:

• Genetics
• Biotechnology
• Medical Research

Background:

• Rare genetic diseases affect millions in India, posing significant healthcare challenges.
• These often fatal disorders result from single-gene mutations, necessitating targeted therapies.
• India's large population underscores the urgent need for effective rare disease management.

Approach:

• This review examines the current landscape of rare genetic diseases in India.
• It analyzes national policies, challenges, and the potential of CRISPR-Cas9 therapies.
• Ethical, regulatory, and accessibility issues of CRISPR are critically evaluated.

Key Points:

• CRISPR-Cas9 technology enables precise correction of disease-causing mutations.
• It holds promise for personalized and effective treatments for rare genetic disorders.
• Addressing ethical and regulatory hurdles is crucial for successful CRISPR implementation.

Conclusions:

• CRISPR-based therapies present a transformative potential for rare genetic diseases in India.
• A multidisciplinary approach integrating science, ethics, and regulation is essential.
• Overcoming challenges will pave the way for innovative healthcare solutions for rare diseases.