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Updated: Aug 11, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Silencing neurodegenerative disease: bringing RNA interference to the clinic
Edgardo Rodriguez-Lebron1, Pedro Gonzalez-Alegre
1Department of Neurology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242-1101, USA. edgardo-rodriguez@mail.medicine.uiowa.edu
Abstract:
RNA interference (RNAi) is a recently described conserved biological pathway where non-coding RNAs suppress the expression of specific genes. Research efforts in the RNAi field aim to gain a better understanding of how its underlying machinery is orchestrated, to define the biological role of this conserved pathway, determine how to effectively manipulate RNAi in the laboratory and to integrate all this knowledge to develop novel therapies for human disease. This review summarizes the advances in the design of therapeutic RNAi for neurodegenerative diseases and discusses some of the experimental steps required to bring this therapy to human clinical trials.
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