Targeting noncoding RNAs in disease
Brian D. Adams,1,2 Christine Parsons,3 Lisa Walker,4 Wen Cai Zhang,4 and Frank J. Slack4
1The RNA Institute, University at Albany, State University of New York, Albany, New York, USA.
2Investigative Medicine Program, Yale University Medical School, New Haven, Connecticut, USA.
3Department of Biochemistry, Bowdoin College, Brunswick, Maine, USA.
4Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Brian D. Adams, Research Faculty, The RNA Institute, University at Albany, State University of New York, 1400 Washington Avenue, Albany, New York 12222, USA. Or to: Brian D. Adams, Investigative Medicine Program, Yale University Medical School, 2 Church Street South, Suite 114, New Haven, Connecticut 06520, USA. Phone: 518.437.4447; E-mail: brian.adams@yale.edu. Or to: Frank J. Slack, Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, 330 Brookline Avenue, Boston, Massachusetts 02115, USA. Phone: 617.735.2601; E-mail: fslack@bidmc.harvard.edu.
Authorship note: B.D. Adams and C. Parsons contributed equally to this work.
Find articles by Adams, B. in: JCI | PubMed | Google Scholar
1The RNA Institute, University at Albany, State University of New York, Albany, New York, USA.
2Investigative Medicine Program, Yale University Medical School, New Haven, Connecticut, USA.
3Department of Biochemistry, Bowdoin College, Brunswick, Maine, USA.
4Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Brian D. Adams, Research Faculty, The RNA Institute, University at Albany, State University of New York, 1400 Washington Avenue, Albany, New York 12222, USA. Or to: Brian D. Adams, Investigative Medicine Program, Yale University Medical School, 2 Church Street South, Suite 114, New Haven, Connecticut 06520, USA. Phone: 518.437.4447; E-mail: brian.adams@yale.edu. Or to: Frank J. Slack, Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, 330 Brookline Avenue, Boston, Massachusetts 02115, USA. Phone: 617.735.2601; E-mail: fslack@bidmc.harvard.edu.
Authorship note: B.D. Adams and C. Parsons contributed equally to this work.
Find articles by Parsons, C. in: JCI | PubMed | Google Scholar
1The RNA Institute, University at Albany, State University of New York, Albany, New York, USA.
2Investigative Medicine Program, Yale University Medical School, New Haven, Connecticut, USA.
3Department of Biochemistry, Bowdoin College, Brunswick, Maine, USA.
4Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Brian D. Adams, Research Faculty, The RNA Institute, University at Albany, State University of New York, 1400 Washington Avenue, Albany, New York 12222, USA. Or to: Brian D. Adams, Investigative Medicine Program, Yale University Medical School, 2 Church Street South, Suite 114, New Haven, Connecticut 06520, USA. Phone: 518.437.4447; E-mail: brian.adams@yale.edu. Or to: Frank J. Slack, Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, 330 Brookline Avenue, Boston, Massachusetts 02115, USA. Phone: 617.735.2601; E-mail: fslack@bidmc.harvard.edu.
Authorship note: B.D. Adams and C. Parsons contributed equally to this work.
Find articles by Walker, L. in: JCI | PubMed | Google Scholar
1The RNA Institute, University at Albany, State University of New York, Albany, New York, USA.
2Investigative Medicine Program, Yale University Medical School, New Haven, Connecticut, USA.
3Department of Biochemistry, Bowdoin College, Brunswick, Maine, USA.
4Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Brian D. Adams, Research Faculty, The RNA Institute, University at Albany, State University of New York, 1400 Washington Avenue, Albany, New York 12222, USA. Or to: Brian D. Adams, Investigative Medicine Program, Yale University Medical School, 2 Church Street South, Suite 114, New Haven, Connecticut 06520, USA. Phone: 518.437.4447; E-mail: brian.adams@yale.edu. Or to: Frank J. Slack, Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, 330 Brookline Avenue, Boston, Massachusetts 02115, USA. Phone: 617.735.2601; E-mail: fslack@bidmc.harvard.edu.
Authorship note: B.D. Adams and C. Parsons contributed equally to this work.
Find articles by Zhang, W. in: JCI | PubMed | Google Scholar
1The RNA Institute, University at Albany, State University of New York, Albany, New York, USA.
2Investigative Medicine Program, Yale University Medical School, New Haven, Connecticut, USA.
3Department of Biochemistry, Bowdoin College, Brunswick, Maine, USA.
4Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Massachusetts, USA.
Address correspondence to: Brian D. Adams, Research Faculty, The RNA Institute, University at Albany, State University of New York, 1400 Washington Avenue, Albany, New York 12222, USA. Or to: Brian D. Adams, Investigative Medicine Program, Yale University Medical School, 2 Church Street South, Suite 114, New Haven, Connecticut 06520, USA. Phone: 518.437.4447; E-mail: brian.adams@yale.edu. Or to: Frank J. Slack, Institute for RNA Medicine, Department of Pathology, Cancer Center, Beth Israel Deaconess Medical Center, Harvard Medical School, 330 Brookline Avenue, Boston, Massachusetts 02115, USA. Phone: 617.735.2601; E-mail: fslack@bidmc.harvard.edu.
Authorship note: B.D. Adams and C. Parsons contributed equally to this work.
Find articles by Slack, F. in: JCI | PubMed | Google Scholar
First published March 1, 2017 - More info
J Clin Invest. 2017;127(3):761–771. doi:10.1172/JCI84424.
Copyright © 2017, American Society for Clinical Investigation
Many RNA species have been identified as important players in the development of chronic diseases, including cancer. Over the past decade, numerous studies have highlighted how regulatory RNAs such as microRNAs (miRNAs) and long noncoding RNAs (lncRNAs) play crucial roles in the development of a disease state. It is clear that the aberrant expression of miRNAs promotes tumor initiation and progression, is linked with cardiac dysfunction, allows for the improper physiological response in maintaining glucose and insulin levels, and can prevent the appropriate integration of neuronal networks, resulting in neurodegenerative disorders. Because of this, there has been a major effort to therapeutically target these noncoding RNAs. In just the past 5 years, over 100 antisense oligonucleotide–based therapies have been tested in phase I clinical trials, a quarter of which have reached phase II/III. Most notable are fomivirsen and mipomersen, which have received FDA approval to treat cytomegalovirus retinitis and high blood cholesterol, respectively. The continued improvement of innovative RNA modifications and delivery entities, such as nanoparticles, will aid in the development of future RNA-based therapeutics for a broader range of chronic diseases. Here we summarize the latest promises and challenges of targeting noncoding RNAs in disease.
- Purchase this article
- $10
- Purchasing this article will give you full access for the calendar year.
- Purchase Site Pass
- $25
- This will give you access to every article on the site for 24 hours.
- Online subscription
- $95
- Individual online subscriptions give you full online access for the calendar year.
- Print subscription
- $795
- Individual print subscriptions give you the print journal and full online access for the year.
- JCI This Month subscription
- $125
- JCI This Month is a 16- to 20-page overview of the articles published each month
- Subscribing to JCI This Month also gives subscribers full online access for the calendar year.
- *Price outside U.S. and Canada: $225.
Copyright © 2017 American Society for Clinical Investigation
ISSN: 0021-9738 (print), 1558-8238 (online)