Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis.
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| Title: | Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis. |
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| Authors: | Grogan M; Cardiovascular Medicine Department, Mayo Clinic, Rochester, MN 55905, USA., Sheikh FH; MedStar Heart and Vascular Institute, MedStar Health/Georgetown University School of Medicine, Washington, DC 20007, USA., Sperry BW; Saint Luke's Mid America Heart Institute, Kansas City, MO 64111, USA., Sweetser MT; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Capocelli K; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Roblin S; Alnylam Pharmaceuticals, Cambridge, MA 02142, USA., Witteles R; Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA 94305, USA. |
| Source: | Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2025 Jun 10; Vol. 36 (3), pp. 102590. Date of Electronic Publication: 2025 Jun 10 (Print Publication: 2025). |
| Publication Type: | Journal Article; Review |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
| ISSN: | 2162-2531 |
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| DOI: | 10.1016/j.omtn.2025.102590 |
