Editing RNA (section)

== Medical applications ==
RNA, initially deemed unsuitable for therapeutics due to its short half-life, has been made useful through advances in stabilization. Therapeutic applications arise as RNA folds into complex conformations and binds proteins, nucleic acids, and small molecules to form catalytic centers.<ref>{{Cite journal |last1=Cech |first1=Thomas R. |last2=Steitz |first2=Joan A. |date=March 2014 |title=The Noncoding RNA Revolution—Trashing Old Rules to Forge New Ones |journal=Cell |volume=157 |issue=1 |pages=77–94 |doi=10.1016/j.cell.2014.03.008 |issn=0092-8674 |pmid=24679528 |s2cid=14852160 |doi-access=free}}</ref> RNA-based vaccines are thought to be easier to produce than traditional vaccines derived from killed or altered pathogens, because it can take months or years to grow and study a pathogen and determine which molecular parts to extract, inactivate, and use in a vaccine. Small molecules with conventional therapeutic properties can target RNA and DNA structures, thereby treating novel diseases. However, research is scarce on small molecules targeting RNA and approved drugs for human illness. Ribavirin, branaplam, and ataluren are currently available medications that stabilize double-stranded RNA structures and control splicing in a variety of disorders.<ref>{{Cite journal |last1=Palacino |first1=James |last2=Swalley |first2=Susanne E |last3=Song |first3=Cheng |last4=Cheung |first4=Atwood K |last5=Shu |first5=Lei |last6=Zhang |first6=Xiaolu |last7=Van Hoosear |first7=Mailin |last8=Shin |first8=Youngah |last9=Chin |first9=Donovan N |last10=Keller |first10=Caroline Gubser |last11=Beibel |first11=Martin |last12=Renaud |first12=Nicole A |last13=Smith |first13=Thomas M |last14=Salcius |first14=Michael |last15=Shi |first15=Xiaoying |date=2015-06-01 |title=SMN2 splice modulators enhance U1–pre-mRNA association and rescue SMA mice |url=|journal=Nature Chemical Biology |volume=11 |issue=7 |pages=511–517 |doi=10.1038/nchembio.1837 |issn=1552-4450 |pmid=26030728}}</ref><ref>{{Cite journal |last1=Roy |first1=Bijoyita |last2=Friesen |first2=Westley J. |last3=Tomizawa |first3=Yuki |last4=Leszyk |first4=John D. |last5=Zhuo |first5=Jin |last6=Johnson |first6=Briana |last7=Dakka |first7=Jumana |last8=Trotta |first8=Christopher R. |last9=Xue |first9=Xiaojiao |last10=Mutyam |first10=Venkateshwar |last11=Keeling |first11=Kim M. |last12=Mobley |first12=James A. |last13=Rowe |first13=Steven M. |last14=Bedwell |first14=David M. |last15=Welch |first15=Ellen M. |date=2016-10-04 |title=Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression |journal=Proceedings of the National Academy of Sciences |volume=113 |issue=44 |pages=12508–12513 |bibcode=2016PNAS..11312508R |doi=10.1073/pnas.1605336113 |issn=0027-8424 |pmc=5098639 |pmid=27702906 |doi-access=free}}</ref> 

Protein-coding mRNAs have emerged as new therapeutic candidates, with RNA replacement being particularly beneficial for brief but torrential protein expression.<ref name="dx.doi.org">{{Cite journal |last1=Qadir |first1=Muhammad Imran |last2=Bukhat |first2=Sherien |last3=Rasul |first3=Sumaira |last4=Manzoor |first4=Hamid |last5=Manzoor |first5=Majid |date=2019-09-03 |title=RNA therapeutics: Identification of novel targets leading to drug discovery |url=|journal=Journal of Cellular Biochemistry |volume=121 |issue=2 |pages=898–929 |doi=10.1002/jcb.29364 |issn=0730-2312 |pmid=31478252 |s2cid=201806158}}</ref> In vitro transcribed mRNAs (IVT-mRNA) have been used to deliver proteins for bone regeneration, pluripotency, and heart function in animal models.<ref>{{Cite journal |last1=Balmayor |first1=Elizabeth R. |last2=Geiger |first2=Johannes P. |last3=Aneja |first3=Manish K. |last4=Berezhanskyy |first4=Taras |last5=Utzinger |first5=Maximilian |last6=Mykhaylyk |first6=Olga |last7=Rudolph |first7=Carsten |last8=Plank |first8=Christian |date=May 2016 |title=Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats |url=|journal=Biomaterials |volume=87 |pages=131–146 |doi=10.1016/j.biomaterials.2016.02.018 |issn=0142-9612 |pmid=26923361}}</ref><ref>{{Cite journal |last1=Plews |first1=Jordan R. |last2=Li |first2=JianLiang |last3=Jones |first3=Mark |last4=Moore |first4=Harry D. |last5=Mason |first5=Chris |last6=Andrews |first6=Peter W. |last7=Na |first7=Jie |date=2010-12-30 |title=Activation of Pluripotency Genes in Human Fibroblast Cells by a Novel mRNA Based Approach |journal=PLOS ONE |volume=5 |issue=12 |pages=e14397 |bibcode=2010PLoSO...514397P |doi=10.1371/journal.pone.0014397 |issn=1932-6203 |pmc=3012685 |pmid=21209933 |doi-access=free}}</ref><ref>{{Cite journal |last1=Preskey |first1=David |last2=Allison |first2=Thomas F. |last3=Jones |first3=Mark |last4=Mamchaoui |first4=Kamel |last5=Unger |first5=Christian |date=May 2016 |title=Synthetically modified mRNA for efficient and fast human iPS cell generation and direct transdifferentiation to myoblasts |url=|journal=Biochemical and Biophysical Research Communications |volume=473 |issue=3 |pages=743–751 |doi=10.1016/j.bbrc.2015.09.102 |issn=0006-291X |pmid=26449459}}</ref><ref>{{Cite journal |last1=Warren |first1=Luigi |last2=Manos |first2=Philip D. |last3=Ahfeldt |first3=Tim |last4=Loh |first4=Yuin-Han |last5=Li |first5=Hu |last6=Lau |first6=Frank |last7=Ebina |first7=Wataru |last8=Mandal |first8=Pankaj K. |last9=Smith |first9=Zachary D. |last10=Meissner |first10=Alexander |last11=Daley |first11=George Q. |last12=Brack |first12=Andrew S. |last13=Collins |first13=James J. |last14=Cowan |first14=Chad |last15=Schlaeger |first15=Thorsten M. |date=November 2010 |title=Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA |url=|journal=Cell Stem Cell |volume=7 |issue=5 |pages=618–630 |doi=10.1016/j.stem.2010.08.012 |issn=1934-5909 |pmc=3656821 |pmid=20888316}}</ref><ref>{{Cite journal |last1=Elangovan |first1=Satheesh |last2=Khorsand |first2=Behnoush |last3=Do |first3=Anh-Vu |last4=Hong |first4=Liu |last5=Dewerth |first5=Alexander |last6=Kormann |first6=Michael |last7=Ross |first7=Ryan D. |last8=Rick Sumner |first8=D. |last9=Allamargot |first9=Chantal |last10=Salem |first10=Aliasger K. |date=November 2015 |title=Chemically modified RNA activated matrices enhance bone regeneration |url=|journal=Journal of Controlled Release |volume=218 |pages=22–28 |doi=10.1016/j.jconrel.2015.09.050 |issn=0168-3659 |pmc=4631704 |pmid=26415855}}</ref> SiRNAs, short RNA molecules, play a crucial role in innate defense against viruses and chromatin structure. They can be artificially introduced to silence specific genes, making them valuable for gene function studies, therapeutic target validation, and drug development.<ref name="dx.doi.org" />

[[mRNA vaccines]] have emerged as an important new class of vaccines, using mRNA to manufacture proteins which provoke an immune response. Their first successful large-scale application came in the form of [[COVID-19 vaccine]]s during the [[COVID-19 pandemic]].