【摘 要】
：
RNA interference is an endogenous cellular mechanism for controlling gene expression in which small interfering RNAs (siRNAs) bind RNA-induced silencing com
【出 处】
：
The 3rd Canton Nucleic Acids Forum(第三届广州核酸国际论坛)
论文部分内容阅读
RNA interference is an endogenous cellular mechanism for controlling gene expression in which small interfering RNAs (siRNAs) bind RNA-induced silencing complex (RISC) to mediate the cleavage of target mRNA.Conjugation of siRNA to an asialoglycoprotein receptor ligand derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery to hepatocytes and silencing of target mRNAs in vitro and in vivo after subcutaneous (SC) administration.Enhanced stabilization chemistry (ESC) of siRNA results in improved potency and duration of action, enabling infrequent, low volume dosing (Nair et al.J.Am.Chem.Soc., 2014).This delivery platform is being employed in several of Alnylams programs, including programs in clinical development.Case studies highlighting preclinical to clinical translation will be presented for genetic medicine programs in TTR-Amyloidosis, Hemophilia and Rare Bleeding Disorders, Hepatic Porphirias and Complement-Mediated Diseases.Lastly, the ESC GalNAc RNAi therapeutic platform is being applied to the identification and development of ALN-HBV for the treatment of chronic hepatitis B.
其他文献
Recent studies came up with novel RNAi triggering molecular structures with unique structural features and functional advantages compared with the conventio
Sample prep plays critical role in modern molecular testing, which is striving for high content and high specificity, to ensure high and unbiased recovery.W
To expand our understanding on how the ribosome functions in the various steps of protein synthesis, we have determined the structures of the 70S ribosome b
Demonstration of a phage-derived peptidic self-assembly targeting two receptors in different geometries Peptide complexation with siRNA forming different ge
Second generation MOE Gapmer antisense oligonucleotides have demonstrated great promise and clinical activity when employed as a therapeutic modality to tar
Next generation sequencing is the breakthrough technology that promotes big data driven approaches in drug discovery research.Several examples of successful
Gene-modified human cells and mice are important tools for studying gene function in vivo.Before 2010, gene-modified human cells and mice were mainly genera