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<p>Sickle cell disease (SCD) is a critical monogenic disorder impacting millions of people in India and severely compromising the quality of life for the patients. At present, the only available curative treatment for sickle cell disease (SCD) is allogenic hematopoietic stem cell transplantation. However, recent advancements in gene therapy offer promising prospects for curing the disease, either through the correction of the pathogenic mutation or the induction of Fetal hemoglobin production. This review focuses on the gene addition and gene editing technologies being currently employed in the treatment of SCD, with a particular emphasis on therapeutic interventions that are undergoing clinical trials globally. Furthermore, this review provides an insight into the extensive research efforts being undertaken in India to develop curative therapies for SCD, while also addressing the significant challenges faced by the healthcare system and patients, including ethical, socio-economic, and regulatory barriers unique to the Indian context.</p>

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

  • 期刊论文

  • 发表日期：

    2025年7月31日

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  • Arushi Goel,
  • Varsha Dalal,
  • Rishi Dhawan,
  • Souvik Maiti,
  • Debojyoti Chakraborty

摘要

Sickle cell disease (SCD) is a critical monogenic disorder impacting millions of people in India and severely compromising the quality of life for the patients. At present, the only available curative treatment for sickle cell disease (SCD) is allogenic hematopoietic stem cell transplantation. However, recent advancements in gene therapy offer promising prospects for curing the disease, either through the correction of the pathogenic mutation or the induction of Fetal hemoglobin production. This review focuses on the gene addition and gene editing technologies being currently employed in the treatment of SCD, with a particular emphasis on therapeutic interventions that are undergoing clinical trials globally. Furthermore, this review provides an insight into the extensive research efforts being undertaken in India to develop curative therapies for SCD, while also addressing the significant challenges faced by the healthcare system and patients, including ethical, socio-economic, and regulatory barriers unique to the Indian context.