JOURNAL ARTICLE

Next-generation therapeutics for rare genetic disorders.

  • Published In: Mutagenesis, 2024, v. 39, n. 3. P. 157 1 of 3

  • Database: Academic Search Ultimate 2 of 3

  • Authored By: Sankar, Akhila; S, Ravi Kumar Y; Singh, Anjali; Roy, Riya; Shukla, Rashmi; Verma, Bhupendra 3 of 3

Abstract

This article focuses on the development and therapeutic potential of next-generation nucleic acid-based therapeutics for rare genetic disorders. It reviews advanced technologies such as CRISPR-Cas9, antisense oligonucleotides (ASOs), small interfering RNA (siRNA), microRNA (miRNA), and aptamers, highlighting their mechanisms, clinical applications, and FDA-approved treatments for diseases including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis, hemophilia, and transthyretin amyloidosis. The article also discusses challenges related to delivery systems, safety, efficacy, and manufacturing, emphasizing the need for improved stability, targeted delivery, and personalized approaches such as n-of-1 trials. Overall, it underscores the promise of these molecular therapies in achieving long-lasting gene modification or replacement while acknowledging ongoing hurdles before widespread clinical adoption.

Additional Information

  • Source:Mutagenesis. 2024/05, Vol. 39, Issue 3, p157
  • Document Type:Article
  • Subject Area:Sports and Leisure
  • Publication Date:2024
  • ISSN:0267-8357
  • DOI:10.1093/mutage/geae002
  • Accession Number:176806302
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