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Progress in RNA-Targeted Therapeutics for Human Diseases
Wangzheqi Zhang1,2,3, Aimin Jiang4,5, Bin-Kui Jia6
1Department of Urology Changzheng Hospital, Naval Medical University (Second Military Medical University) Shanghai China.
Medcomm
|February 9, 2026
Summary
RNA therapeutics, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA), offer new ways to treat diseases by targeting RNA. Advances in delivery and design are overcoming challenges for wider clinical use.
Area of Science:
- Molecular Medicine
- RNA Biology
- Therapeutics Development
Background:
- RNA's role extends beyond genetics to regulation, signaling, and epigenetics.
- The traditional protein-centric view is shifting to an RNA-regulatory network paradigm.
- Diverse RNA therapeutics are emerging due to advances in sequencing, structural biology, and delivery.
Purpose of the Study:
- To provide a concise comparison of major RNA therapeutic modalities.
- To outline mechanisms and applications of RNA-based strategies.
- To discuss translational barriers and potential solutions for RNA therapeutics.
Main Methods:
- Review of current literature on RNA therapeutics.
- Analysis of mechanisms for gene silencing, editing, protein replacement, immune activation, and drug delivery.
- Discussion of challenges and advancements in RNA therapeutic development.
Main Results:
- Detailed overview of RNA modalities like ASOs, siRNAs, miRNA modulators, mRNA therapeutics, aptamers, shRNA, and CRISPR/Cas.
- Emphasis on ASOs and siRNAs for neurological, metabolic, and infectious diseases.
- Highlighting mRNA therapeutics in vaccine development and their potential.
Conclusions:
- RNA therapeutics hold significant promise for precision medicine.
- Overcoming challenges in stability, delivery, and immunogenicity is crucial for clinical translation.
- Chemical modifications, nanotechnology, and AI-assisted design are key to advancing next-generation RNA therapies.
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