Unveiling the potential of antisense oligonucleotides: Mechanisms, therapies, and safety insights

Ersöz E., DEMİR DORA D.

Drug Development Research, cilt.85, sa.4, 2024 (SCI-Expanded) identifier identifier

  • Yayın Türü: Makale / Derleme
  • Cilt numarası: 85 Sayı: 4
  • Basım Tarihi: 2024
  • Doi Numarası: 10.1002/ddr.22187
  • Dergi Adı: Drug Development Research
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, BIOSIS, Biotechnology Research Abstracts, CAB Abstracts, Chemical Abstracts Core, EMBASE, International Pharmaceutical Abstracts, Veterinary Science Database
  • Anahtar Kelimeler: antisense oligonucleotide, drug, gene therapy, pharmacovigilance, safety
  • Akdeniz Üniversitesi Adresli: Evet

Özet

Antisense oligonucleotides (ASOs) are short, synthetic, single-stranded deoxynucleotide sequences composed of phosphate backbone-connected sugar rings. Designing of those strands is based on Watson-Crick hydrogen bonding mechanism. Thanks to rapidly advancing medicine and technology, evolving of the gene therapy area and ASO approaches gain attention. Considering the genetic basis of diseases, it is promising that gene therapy approaches offer more specific and effective options compared to conventional treatments. The objective of this review is to explain the mechanism of ASOs and discuss the characteristics and safety profiles of therapeutic agents in this field. Pharmacovigilance for gene therapy products is complex, requiring accurate assessment of benefit-risk balance and evaluation of adverse effects.