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Development of siRNA Therapeutics for the Treatment of Liver Diseases.
- Source :
-
Methods in molecular biology (Clifton, N.J.) [Methods Mol Biol] 2021; Vol. 2282, pp. 57-75. - Publication Year :
- 2021
-
Abstract
- Small interfering RNA (siRNA)-based therapeutics holds the promise to treat a wide range of human diseases that are currently incurable using conventional therapies. Most siRNA therapeutic efforts to date have focused on the treatment of liver diseases due to major breakthroughs in the development of efficient strategies for delivering siRNA drugs to the liver. Indeed, the development of lipid nanoparticle-formulated and GalNAc-conjugated siRNA therapeutics has resulted in recent FDA approvals of the first siRNA-based drugs, patisiran for the treatment of hereditary transthyretin amyloidosis and givosiran for the treatment of acute hepatic porphyria, respectively. Here, we describe the current strategies for delivering siRNA drugs to the liver and summarize recent advances in clinical development of siRNA therapeutics for the treatment of liver diseases.
- Subjects :
- Acetylgalactosamine analogs & derivatives
Acetylgalactosamine therapeutic use
Amyloid Neuropathies, Familial genetics
Amyloid Neuropathies, Familial metabolism
Amyloid Neuropathies, Familial therapy
Animals
Gene Transfer Techniques
Humans
Liver Diseases genetics
Liver Diseases metabolism
Porphyrias, Hepatic diagnosis
Porphyrias, Hepatic metabolism
Porphyrias, Hepatic therapy
Pyrrolidines therapeutic use
RNA, Small Interfering genetics
RNA, Small Interfering metabolism
Liver Diseases therapy
RNA Interference
RNA, Small Interfering therapeutic use
RNAi Therapeutics
Subjects
Details
- Language :
- English
- ISSN :
- 1940-6029
- Volume :
- 2282
- Database :
- MEDLINE
- Journal :
- Methods in molecular biology (Clifton, N.J.)
- Publication Type :
- Academic Journal
- Accession number :
- 33928570
- Full Text :
- https://doi.org/10.1007/978-1-0716-1298-9_5