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siRNAs—A New Class of Medicines

To identify the key insights or developments described in this article
1 Credit CME

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.

Article Information

Corresponding Author: Anastasia Khvorova, PhD, RNA Therapeutics Institute, UMass Chan Medical School, 368 Plantation St, Worcester, MA 01605 (anastasia.khvorova@umassmed.edu).

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.

AMA CME Accreditation Information

Credit Designation Statement: The American Medical Association designates this Journal-based CME activity activity for a maximum of 1.00  AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to:

  • 1.00 Medical Knowledge MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program;;
  • 1.00 Self-Assessment points in the American Board of Otolaryngology – Head and Neck Surgery’s (ABOHNS) Continuing Certification program;
  • 1.00 MOC points in the American Board of Pediatrics’ (ABP) Maintenance of Certification (MOC) program;
  • 1.00 Lifelong Learning points in the American Board of Pathology’s (ABPath) Continuing Certification program; and
  • 1.00 credit toward the CME of the American Board of Surgery’s Continuous Certification program

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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