Gene Therapy Yields to RNA Interference
Authors: Jennifer Fisher Wilson
Journal: Annals of Internal Medicine (2005)

Current Clinical Issues19 July 2005Gene Therapy Yields to RNA InterferenceJennifer Fisher WilsonJennifer Fisher WilsonSearch for more papers by this authorAuthor, Article, and Disclosure Informationhttps://doi.org/10.7326/0003-4819-143-2-200507190-00033 SectionsAboutFull TextPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissions ShareFacebookTwitterLinkedInRedditEmail Over the past 2 decades, physicians heard a lot about the promise of gene therapy. Then, suddenly, it stopped. Years of work added up to little more than 1 tough lesson after another on the complexity of therapeutic DNA fixing. Deaths in 1998 and 2002 in 2 gene therapy trials particularly contributed to its fall from favor. In frustration, many researchers and pharmaceutical companies abandoned their efforts altogether.Others turned to alternative gene therapy, focused not on fixing DNA but on blocking its effects. Ribozyme and antisense gene therapy targeted messenger RNA (mRNA) transcripts to prevent reproduction of a mutated ...References1. Harper SQ, Staber PD, He X, et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci. 2005;102:5820-5. [PMID: 15811941] CrossrefMedlineGoogle Scholar2. Soutschek J, Akinc A, Bramlage B, et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNA. Nature. 2004;432:173-8. [PMID: 15538359] CrossrefMedlineGoogle Scholar3. Ling X, Arlinghaus RB. Knockdown of STAT3 expression by RNA interference inhibits the induction of breast tumors in immunocompetent mice. Cancer Res. 2005;65:2532-6. [PMID: 15805244] CrossrefMedlineGoogle Scholar4. Zhang Y, Zhang YF, Bryant J, et al. Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer. Clin Cancer Res. 2004;10:3667-77. [PMID: 15173073] CrossrefMedlineGoogle Scholar5. Ge Q, Filip L, Bai A, et al. Inhibition of influenza virus production in virus-infected mice by RNA interference. Proc Natl Acad Sci U S A. 2004;101:8676-81. [PMID: 15173599] CrossrefMedlineGoogle ScholarFurther Reading6. Hannon GJ. RNA interference. Nature. 2002;418:244-51. [PMID: 12110901] CrossrefMedlineGoogle Scholar7. Hannon GJ, Rossi JJ. Unlocking the potential of the human genome with RNA interference. Nature. 2004;431:371-8. [PMID: 15372045] CrossrefMedlineGoogle Scholar8. Shankar P, Manjunath N, Lieberman J. The prospect of silencing disease using RNA interference. JAMA. 2005;293:1367-73. [PMID: 15769970] CrossrefMedlineGoogle Scholar Author, Article, and Disclosure InformationAffiliations: Disclosures: None disclosed.E-mail:[email protected]org. PreviousarticleNextarticle Advertisement FiguresReferencesRelatedDetails Metrics Cited byThe potential of RNA interference-based therapies for viral infectionsRNA Interference in Neuroscience: Progress and Challenges 19 July 2005Volume 143, Issue 2Page: 161-164KeywordsBrainDNADrugsGene therapyLungsProteinsRNARNA interferenceScientistsSmall interfering RNA ePublished: 19 July 2005 Issue Published: 19 July 2005 Copyright & PermissionsCopyright © 2005 by American College of Physicians. All Rights Reserved.PDF downloadLoading ...