The Limitless Future of RNA Therapeutics.

Autor:	Damase TR; RNA Therapeutics Program, Department of Cardiovascular Sciences, Houston Methodist Research Institute, Houston, TX, United States., Sukhovershin R; RNA Therapeutics Program, Department of Cardiovascular Sciences, Houston Methodist Research Institute, Houston, TX, United States., Boada C; Colleges of Medicine, Engineering, Texas A&M University and Houston Methodist Hospital, Houston, TX, United States., Taraballi F; Center for Musculoskeletal Regeneration, Houston Methodist Research Institute, Houston, TX, United States.; Department of Orthopedics and Sports Medicine, Houston Methodist Hospital, Houston, TX, United States., Pettigrew RI; Colleges of Medicine, Engineering, Texas A&M University and Houston Methodist Hospital, Houston, TX, United States., Cooke JP; RNA Therapeutics Program, Department of Cardiovascular Sciences, Houston Methodist Research Institute, Houston, TX, United States.
Jazyk:	angličtina
Zdroj:	Frontiers in bioengineering and biotechnology [Front Bioeng Biotechnol] 2021 Mar 18; Vol. 9, pp. 628137. Date of Electronic Publication: 2021 Mar 18 (Print Publication: 2021).
DOI:	10.3389/fbioe.2021.628137
Abstrakt:	Recent advances in the generation, purification and cellular delivery of RNA have enabled development of RNA-based therapeutics for a broad array of applications. RNA therapeutics comprise a rapidly expanding category of drugs that will change the standard of care for many diseases and actualize personalized medicine. These drugs are cost effective, relatively simple to manufacture, and can target previously undruggable pathways. It is a disruptive therapeutic technology, as small biotech startups, as well as academic groups, can rapidly develop new and personalized RNA constructs. In this review we discuss general concepts of different classes of RNA-based therapeutics, including antisense oligonucleotides, aptamers, small interfering RNAs, microRNAs, and messenger RNA. Furthermore, we provide an overview of the RNA-based therapies that are currently being evaluated in clinical trials or have already received regulatory approval. The challenges and advantages associated with use of RNA-based drugs are also discussed along with various approaches for RNA delivery. In addition, we introduce a new concept of hospital-based RNA therapeutics and share our experience with establishing such a platform at Houston Methodist Hospital. Competing Interests: Houston Methodist Hospital has been assigned intellectual property related to the synthesis, purification, validation, and delivery of nucleic acid therapeutics. JPC is an inventor on issued patents related to mRNA telomerase therapy, which have been assigned to Stanford University and licensed to his company. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest. (Copyright © 2021 Damase, Sukhovershin, Boada, Taraballi, Pettigrew and Cooke.)
Databáze:	MEDLINE
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