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In recent years, the use of N-acetylgalactosamine (GalNAc) conjugates to target asialoglycoprotein receptors and further deliver oligonucleotides to hepatocytes has become a breakthrough approach in the field of therapeutic oligonucleotides. Compared with traditional delivery methods of nucleic acid drugs, GalNAc-nucleic acid conjugation is a simple method of liver delivery. It exhibits liver targeting specificity, high efficiency, high safety, large-scale preparation and many other advantages, thus has research value of in-depth development and broad prospects for clinical application. Givosiran, developed using GalNAc conjugation, has been approved by FDA for the treatment of acute hepatic porphyria, and there are 7 other conjugates are in registration review or phase III clinical trials, and at least 21 GalNAc nucleic acid conjugates are in the early stages of clinical development. However, the relevant research foundation of GalNAc conjugation in China is relatively weak, and no related products are on the market, making it a neck-stuck technology in China. Therefore, this review focuses on GalNAc and its derivatives as nucleic acid drug delivery vehicles and their application progress in small interfering RNA (siRNA) and antisense oligonucleotides (ASO). This review provides reference for the development of domestic GalNAc conjugates.
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| 科 Family | 属数 Number of genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) | 属 Genus | 种数 Number of species | 占总种数比例 Percentage of total species (%) |
|---|---|---|---|---|---|---|
| 鹅膏菌科Amanitaceae | 2 | 11 | 5.26 | 鹅膏菌属 Amanita | 10 | 4.78 |
| 小菇科 Mycenaceae | 2 | 12 | 5.74 | 丝盖伞属 Inocybe | 5 | 2.39 |
| 多孔菌科 Polyporaceae | 8 | 14 | 6.70 | 蜡蘑属 Laccaria | 5 | 2.39 |
| 红菇科 Russulaceae | 3 | 23 | 11.00 | 小皮伞属 Marasmius | 6 | 2.87 |
| 小菇属 Mycena | 11 | 5.26 | ||||
| 光柄菇属 Pluteus | 5 | 2.39 | ||||
| 红菇属 Russula | 17 | 8.13 | ||||
| 栓菌属 Trametes | 5 | 2.39 |
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