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CRISPR-Cas9 Gene Therapy: Revolutionizing the Treatment of Genetic Disorders

  • Unique Paper ID: 181821
  • Volume: 12
  • Issue: 1
  • PageNo: 5589-5595
  • Abstract:
  • CRISPR-Cas9 has emerged as a groundbreaking genome-editing technology that enables targeted, efficient, and cost-effective modification of genetic material. Originating from a natural bacterial defense system, CRISPR-Cas9 has significantly advanced beyond previous tools such as Zinc Finger Nucleases (ZFNs) and Transcription Activator-Like Effector Nucleases (TALENs), owing to its simplicity, precision, and scalability. The system uses a guide RNA (gRNA) to direct the Cas9 enzyme to specific DNA sequences, where it induces double-strand breaks. These breaks are repaired by cellular mechanisms—non-homologous end joining (NHEJ) or homology-directed repair (HDR) allowing for gene disruption, correction, or insertion. This review explores the mechanism of CRISPR-Cas9 and highlights its therapeutic applications in various genetic disorders, including sickle cell anemia, cystic fibrosis, Huntington’s disease, and Leber congenital amaurosis. In several of these conditions, CRISPR-based therapies have shown promising results in clinical and preclinical studies, leading to breakthroughs such as the FDA-approved Casgevy for sickle cell disease. Emerging technologies like base and prime editing enhance precision by enabling nucleotide-level changes without inducing double-strand breaks. Despite its potential, CRISPR faces significant challenges including off-target effects, delivery limitations, and immune responses. Ethical concerns, particularly regarding germline editing and equitable access, also demand careful regulation and societal dialogue.
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Cite This Article

  • ISSN: 2349-6002
  • Volume: 12
  • Issue: 1
  • PageNo: 5589-5595

CRISPR-Cas9 Gene Therapy: Revolutionizing the Treatment of Genetic Disorders

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Journal no 47859

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