The Limitless Future of RNA Therapeutics
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 per...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2021-03-01
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| Series: | Frontiers in Bioengineering and Biotechnology |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fbioe.2021.628137/full |
| _version_ | 1831527301499584512 |
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| author | Tulsi Ram Damase Roman Sukhovershin Christian Boada Francesca Taraballi Francesca Taraballi Roderic I. Pettigrew John P. Cooke |
| author_facet | Tulsi Ram Damase Roman Sukhovershin Christian Boada Francesca Taraballi Francesca Taraballi Roderic I. Pettigrew John P. Cooke |
| author_sort | Tulsi Ram Damase |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-12-16T16:26:12Z |
| format | Article |
| id | doaj.art-a499de2a1a0547d4aecc43bb88763c27 |
| institution | Directory Open Access Journal |
| issn | 2296-4185 |
| language | English |
| last_indexed | 2024-12-16T16:26:12Z |
| publishDate | 2021-03-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Bioengineering and Biotechnology |
| spelling | doaj.art-a499de2a1a0547d4aecc43bb88763c272022-12-21T22:24:44ZengFrontiers Media S.A.Frontiers in Bioengineering and Biotechnology2296-41852021-03-01910.3389/fbioe.2021.628137628137The Limitless Future of RNA TherapeuticsTulsi Ram Damase0Roman Sukhovershin1Christian Boada2Francesca Taraballi3Francesca Taraballi4Roderic I. Pettigrew5John P. Cooke6RNA Therapeutics Program, Department of Cardiovascular Sciences, Houston Methodist Research Institute, Houston, TX, United StatesRNA Therapeutics Program, Department of Cardiovascular Sciences, Houston Methodist Research Institute, Houston, TX, United StatesColleges of Medicine, Engineering, Texas A&M University and Houston Methodist Hospital, Houston, TX, United StatesCenter for Musculoskeletal Regeneration, Houston Methodist Research Institute, Houston, TX, United StatesDepartment of Orthopedics and Sports Medicine, Houston Methodist Hospital, Houston, TX, United StatesColleges of Medicine, Engineering, Texas A&M University and Houston Methodist Hospital, Houston, TX, United StatesRNA Therapeutics Program, Department of Cardiovascular Sciences, Houston Methodist Research Institute, Houston, TX, United StatesRecent 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.https://www.frontiersin.org/articles/10.3389/fbioe.2021.628137/fullRNA therapeuticsdelivery of RNA therapeuticshospital-based RNA therapeuticsmessenger RNAs (mRNAs)self-amplifying mRNA |
| spellingShingle | Tulsi Ram Damase Roman Sukhovershin Christian Boada Francesca Taraballi Francesca Taraballi Roderic I. Pettigrew John P. Cooke The Limitless Future of RNA Therapeutics Frontiers in Bioengineering and Biotechnology RNA therapeutics delivery of RNA therapeutics hospital-based RNA therapeutics messenger RNAs (mRNAs) self-amplifying mRNA |
| title | The Limitless Future of RNA Therapeutics |
| title_full | The Limitless Future of RNA Therapeutics |
| title_fullStr | The Limitless Future of RNA Therapeutics |
| title_full_unstemmed | The Limitless Future of RNA Therapeutics |
| title_short | The Limitless Future of RNA Therapeutics |
| title_sort | limitless future of rna therapeutics |
| topic | RNA therapeutics delivery of RNA therapeutics hospital-based RNA therapeutics messenger RNAs (mRNAs) self-amplifying mRNA |
| url | https://www.frontiersin.org/articles/10.3389/fbioe.2021.628137/full |
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