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Updated: Apr 23, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
mRNA-based therapeutics--developing a new class of drugs
Ugur Sahin1, Katalin Karikó2, Özlem Türeci3
11] TRON Translational Oncology at the University Medical Center of the Johannes Gutenberg University, Langenbeckstrasse 1, 55131 Mainz, Germany. [2] BioNTech Corporation, An der Goldgrube 12, 55131 Mainz, Germany.
Synthetic messenger RNA (mRNA) is a promising new drug class for delivering genetic information. Advances in controlling mRNA
Area of Science:
- Biotechnology
- Molecular Biology
- Drug Discovery
Background:
- In vitro transcribed (IVT) mRNA is emerging as a novel drug class.
- Synthetic mRNA mimics natural mRNA for transient protein expression.
- Key challenges include controlling translational efficacy and immunogenicity.
Purpose of the Study:
- To provide a comprehensive overview of mRNA-based drug technologies.
- To discuss current applications and future potential of IVT mRNA.
- To identify key challenges and opportunities in mRNA drug development.
Main Methods:
- Review of current literature on IVT mRNA.
- Analysis of advancements in mRNA technology.
- Discussion of emerging applications and challenges.
Main Results:
- IVT mRNA drug technology is rapidly advancing.
- Clinical development includes cancer immunotherapies and vaccines.
- Novel applications include in vivo protein replacement and genome engineering.
Conclusions:
- IVT mRNA represents a versatile new drug class.
- Significant progress has been made in overcoming technical hurdles.
- Further development holds potential for diverse therapeutic applications.
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