Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics
2016; Cell Press; Volume: 6; Linguagem: Inglês
10.1016/j.omtn.2016.12.003
ISSN2162-2531
Autores Tópico(s)Virus-based gene therapy research
ResumoA main challenge in realizing the full potential of nucleic acid therapeutics is efficient delivery of them into targeted tissues and cells. N-acetylgalactosamine (GalNAc) is a well-defined liver-targeted moiety benefiting from its high affinity with asialoglycoprotein receptor (ASGPR). By conjugating it directly to the oligonucleotides or decorating it to a certain delivery system as a targeting moiety, GalNAc has achieved compelling successes in the development of nucleic acid therapeutics in recent years. Several oligonucleotide modalities are undergoing pivotal clinical studies, followed by a blooming pipeline in the preclinical stage. This review covers the progress of GalNAc-decorated oligonucleotide drugs, including siRNAs, anti-miRs, and ASOs, which provides a panorama for this field.
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