Small Interfering RNA (siRNA) Therapy: Gene Therapy for Inherited Diseases-An Updated Review for Healthcare Professionals

Document Type : Review Articles

Authors

Ministry of National Guard, Saudi Arabia

Abstract

Background: Small interfering RNA (siRNA) medicines have progressed from proof-of-concept to clinical reality, culminating in six U.S. FDA-approved agents that silence hepatic transcripts driving amyloid neuropathy, porphyria, hyperoxaluria, and atherogenic dyslipidemia.

Aim: To synthesize up-to-date knowledge on the therapeutic class, detailing mechanisms, indications, dosing, safety, and monitoring.

Methods: Structured narrative review of the supplied article integrating mechanistic principles of RNA interference, regimen specifics, adverse-event profiles, contraindications, and practice-based monitoring.

Results: Patisiran (LNP-delivered) and GalNAc-conjugated givosiran, lumasiran, inclisiran, nedosiran, and vutrisiran exploit dicer/RISC-mediated cleavage of target mRNAs (TTR, ALAS1, HAO1, PCSK9, LDHA), achieving durable protein knockdown. Administration spans intravenous infusion (patisiran) and infrequent subcutaneous regimens (others), with weight-based loading for select pediatric indications. Class-specific risks include infusion reactions (patisiran) and injection-site reactions; agent-specific signals include transaminase elevation, renal indices and hyperhomocysteinemia (givosiran), and vitamin A depletion requiring supplementation (vutrisiran). Hypersensitivity to givosiran is an absolute contraindication.

Conclusion: siRNA therapeutics deliver mechanism-based control of diverse inherited and cardiometabolic diseases through precise, durable transcript silencing; safe implementation hinges on route-appropriate administration, proactive laboratory surveillance, nutritional stewardship where relevant, and standardized algorithms for missed doses and dose modification

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Egyptian Journal of Chemistry
Volume 68, Issue 13 - Serial Number 13
(In Loving Memory of Late Professor Doctor”Zeinab M. Nofal” In progress
December 2025
Pages 1235-1246

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History

  • Receive Date: 08 August 2025
  • Revise Date: 28 September 2025
  • Accept Date: 02 October 2025

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