دورية أكاديمية
Engineering approaches for RNA-based and cell-based osteoarthritis therapies.
| العنوان: | Engineering approaches for RNA-based and cell-based osteoarthritis therapies. |
|---|---|
| المؤلفون: | DeJulius CR; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA., Walton BL; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA., Colazo JM; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA., d'Arcy R; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA., Francini N; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA., Brunger JM; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA. jonathan.m.brunger@vanderbilt.edu., Duvall CL; Department of Biomedical Engineering, Vanderbilt University, Nashville, TN, USA. craig.duvall@vanderbilt.edu. |
| المصدر: | Nature reviews. Rheumatology [Nat Rev Rheumatol] 2024 Feb; Vol. 20 (2), pp. 81-100. Date of Electronic Publication: 2024 Jan 22. |
| نوع المنشور: | Journal Article; Review |
| اللغة: | English |
| بيانات الدورية: | Publisher: Nature Pub. Group Country of Publication: United States NLM ID: 101500080 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1759-4804 (Electronic) Linking ISSN: 17594790 NLM ISO Abbreviation: Nat Rev Rheumatol Subsets: MEDLINE |
| أسماء مطبوعة: | Original Publication: New York, NY : Nature Pub. Group |
| مواضيع طبية MeSH: | CRISPR-Cas Systems* , Osteoarthritis*/genetics , Osteoarthritis*/therapy, Humans ; RNA ; Quality of Life ; Gene Editing |
| مستخلص: | Osteoarthritis (OA) is a chronic, debilitating disease that substantially impairs the quality of life of affected individuals. The underlying mechanisms of OA are diverse and are becoming increasingly understood at the systemic, tissue, cellular and gene levels. However, the pharmacological therapies available remain limited, owing to drug delivery barriers, and consist mainly of broadly immunosuppressive regimens, such as corticosteroids, that provide only short-term palliative benefits and do not alter disease progression. Engineered RNA-based and cell-based therapies developed with synthetic chemistry and biology tools provide promise for future OA treatments with durable, efficacious mechanisms of action that can specifically target the underlying drivers of pathology. This Review highlights emerging classes of RNA-based technologies that hold potential for OA therapies, including small interfering RNA for gene silencing, microRNA and anti-microRNA for multi-gene regulation, mRNA for gene supplementation, and RNA-guided gene-editing platforms such as CRISPR-Cas9. Various cell-engineering strategies are also examined that potentiate disease-dependent, spatiotemporally regulated production of therapeutic molecules, and a conceptual framework is presented for their application as OA treatments. In summary, this Review highlights modern genetic medicines that have been clinically approved for other diseases, in addition to emerging genome and cellular engineering approaches, with the goal of emphasizing their potential as transformative OA treatments. (© 2024. Springer Nature Limited.) |
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| معلومات مُعتمدة: | R21 AR079245 United States AR NIAMS NIH HHS; R21 AR079683 United States AR NIAMS NIH HHS; T32 EB021937 United States EB NIBIB NIH HHS; R01 AR078666 United States AR NIAMS NIH HHS; T32 GM007347 United States GM NIGMS NIH HHS; R21 AR078636 United States AR NIAMS NIH HHS |
| المشرفين على المادة: | 63231-63-0 (RNA) |
| تواريخ الأحداث: | Date Created: 20240122 Date Completed: 20240201 Latest Revision: 20240529 |
| رمز التحديث: | 20240529 |
| مُعرف محوري في PubMed: | PMC11129836 |
| DOI: | 10.1038/s41584-023-01067-4 |
| PMID: | 38253889 |
| قاعدة البيانات: | MEDLINE |
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| DOI: | 10.1038/s41584-023-01067-4 |