Rare genetic diseases are a group of life-threatening disorders affecting significant populations worldwide and posing substantial challenges to healthcare systems globally. India, with its vast population, is also no exception. The country harbors millions of individuals affected by these fatal disorders, which often result from mutations in a single gene. The emergence of CRISPR-Cas9 technology, however, has ushered in a new era of hope in genetic therapies. CRISPR-based treatments hold the potential to precisely edit and correct disease-causing mutations, offering tailored solutions for rare genetic diseases in India. This review explores the landscape of rare genetic diseases in India along with national policies and major challenges, and examines the implications of CRISPR-based therapies for potential cure. It delves into the potential of this technology in providing personalized and effective treatments. However, alongside these promising prospects, some ethical considerations, regulatory challenges, and concerns about the accessibility of CRISPR therapies are also discussed since addressing these issues is crucial for harnessing the full power of CRISPR in tackling rare genetic diseases in India. By taking a multidisciplinary approach that combines scientific advancements, ethical principles, and regulatory frameworks, these complexities can be reconciled, paving the way for innovative and impactful healthcare solutions for rare diseases in India.

罕见遗传病是一组危及生命的疾病，影响全球大量人口，并对全球医疗保健系统构成重大挑战。人口众多的印度也不例外。该国有数百万人受到这些致命疾病的影响，这些疾病通常是由单个基因突变引起的。然而，CRISPR-Cas9技术的出现，为基因治疗带来了希望的新时代。基于 CRISPR 的治疗方法具有精确编辑和纠正致病突变的潜力，为印度罕见遗传病提供量身定制的解决方案。这篇综述探讨了印度罕见遗传病的概况以及国家政策和主要挑战，并探讨了基于 CRISPR 的疗法对潜在治愈方法的影响。它深入研究了该技术在提供个性化和有效治疗方面的潜力。然而，除了这些充满希望的前景之外，还讨论了一些伦理考虑、监管挑战以及对 CRISPR 疗法可及性的担忧，因为解决这些问题对于充分利用 CRISPR 的力量来应对印度的罕见遗传病至关重要。通过采取结合科学进步、伦理原则和监管框架的多学科方法，可以协调这些复杂性，为印度罕见病创新和有效的医疗保健解决方案铺平道路。