Small interfering RNA (siRNA) is one of several nucleic acid molecules representing a third class of therapeutic modalities, in addition to small molecules and biologics. Like small molecules, siRNAs have a defined chemical structure, are inexpensive to manufacture, and are stable at room temperature, but differ in their mechanism of action. By harnessing a fundamental cellular mechanism for turning off genes, siRNAs can downregulate virtually any molecular target with high potency, specificity, and multimonth duration of action.
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CME Disclosure Statement: Unless noted, all individuals in control of content reported no relevant financial relationships. If applicable, all relevant financial relationships have been mitigated.
Corresponding Author: Anastasia Khvorova, PhD, RNA Therapeutics Institute, UMass Chan Medical School, 368 Plantation St, Worcester, MA 01605 (email@example.com).
Published Online: June 12, 2023. doi:10.1001/jama.2023.4570
Conflict of Interest Disclosures: Dr Khvorova reports being an unpaid director at large of American Society of Gene & Cell Therapy and scientific council member of the Oligonucleotide Therapeutics Society; founder and scientific adviser of Atalanta Therapeutics, Comanche Biopharma, and Advirna Genomics; reported grants from the National Institutes of Health (Maximizing Investigators’ Research Award) during the conduct of the study; personal fees (scientific advisory board) from Atalanta, Comanche, and Evox outside the submitted work; and more than 30 patents and applications relevant to RNA interference licensed to Atalanta, Comanche, Aldena, and Voyager. All intellectual property is owned and managed by UMass Chan Medical School.
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Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to:
It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting MOC credit.
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