siRNA therapeutics and its challenges: Recent advances in effective delivery for cancer therapy

Arpita Paul, Anuraag Muralidharan, Avirup Biswas, B. Venkatesh Kamath, Alex Joseph, Angel Treasa Alex

Research output: Contribution to journalReview articlepeer-review

1 Citation (Scopus)


Small interfering RNA (siRNA) has emerged as a promising strategy for treating several “undruggable” genetically impaired conditions. The siRNA-based drugs have opened the doors to a novel mode of therapy. Though siRNA is a potential therapeutic strategy to treat various disorders, its application is still not widely accepted due to certain drawbacks such as off-target effects, stability etc. To overcome these hurdles, several promising modifications and strategies have been reported. Cancer has been identified to have several involvement of genetic imbalances, where siRNA-based formulations have shown promising results both in vitro and in vivo. Apart from cancer, orphan diseases too have an urgent need for novel and effective remedies which is slowly being fulfilled by the chemically synthesized siRNA therapeutics. Till date five siRNA-based drugs namely Patisiran, Givlaari, Oxlumo™, Leqvio and Amvuttra have been approved by USFDA, which have initiated a new category of biopharmaceuticals in the market.

Original languageEnglish
Article number100063
Publication statusPublished - 01-07-2022

All Science Journal Classification (ASJC) codes

  • Internal Medicine
  • Biotechnology
  • Pharmaceutical Science
  • Pharmacology (medical)


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