GalNAc-siRNA conjugates: leading the way for delivery of RNAi therapeutics
AD Springer, SF Dowdy - Nucleic acid therapeutics, 2018 - liebertpub.com
Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide
variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease …
variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease …
Advanced siRNA designs further improve in vivo performance of GalNAc-siRNA conjugates
DJ Foster, CR Brown, S Shaikh, C Trapp, MK Schlegel… - Molecular Therapy, 2018 - cell.com
Significant progress has been made in the advancement of RNAi therapeutics by combining
a synthetic triantennary N-acetylgalactosamine ligand targeting the asialoglycoprotein …
a synthetic triantennary N-acetylgalactosamine ligand targeting the asialoglycoprotein …
Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
JK Nair, JLS Willoughby, A Chan… - Journal of the …, 2014 - ACS Publications
Conjugation of small interfering RNA (siRNA) to an asialoglycoprotein receptor ligand
derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery of the siRNA to …
derived from N-acetylgalactosamine (GalNAc) facilitates targeted delivery of the siRNA to …
Evaluation of GalNAc-siRNA conjugate activity in pre-clinical animal models with reduced asialoglycoprotein receptor expression
JLS Willoughby, A Chan, A Sehgal, JS Butler, JK Nair… - Molecular Therapy, 2018 - cell.com
The hepatocyte-specific asialoglycoprotein receptor (ASGPR) is an ideal candidate for
targeted drug delivery to the liver due to its high capacity for substrate clearance from …
targeted drug delivery to the liver due to its high capacity for substrate clearance from …
siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in …
S Matsuda, K Keiser, JK Nair, K Charisse… - ACS chemical …, 2015 - ACS Publications
Asialoglycoprotein receptor (ASGPR) mediated delivery of triantennary N-
acetylgalactosamine (GalNAc) conjugated short interfering RNAs (siRNAs) to hepatocytes is …
acetylgalactosamine (GalNAc) conjugated short interfering RNAs (siRNAs) to hepatocytes is …
Liver-targeted delivery of oligonucleotides with N-acetylgalactosamine conjugation
H Cui, X Zhu, S Li, P Wang, J Fang - ACS omega, 2021 - ACS Publications
The potential therapeutic application of oligonucleotides (ONs) that selectively suppress
target genes through antisense and RNA interference mechanisms has attracted great …
target genes through antisense and RNA interference mechanisms has attracted great …
Clinical proof of concept for a novel hepatocyte-targeting GalNAc-siRNA conjugate
TS Zimmermann, V Karsten, A Chan, J Chiesa… - Molecular therapy, 2017 - cell.com
Advancement of RNAi-based therapeutics depends on effective delivery to the site of protein
synthesis. Although intravenously administered, multi-component delivery vehicles have …
synthesis. Although intravenously administered, multi-component delivery vehicles have …
Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates
One hallmark of trivalent N-acetylgalactosamine (GalNAc)-conjugated siRNAs is the
remarkable durability of silencing that can persist for months in preclinical species and …
remarkable durability of silencing that can persist for months in preclinical species and …
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates
JK Nair, H Attarwala, A Sehgal, Q Wang… - Nucleic acids …, 2017 - academic.oup.com
Covalent attachment of a synthetic triantennary N-acetylagalactosamine (GalNAc) ligand to
chemically modified siRNA has enabled asialoglycoprotein (ASGPR)-mediated targeted …
chemically modified siRNA has enabled asialoglycoprotein (ASGPR)-mediated targeted …
Preclinical and clinical advances of GalNAc-decorated nucleic acid therapeutics
Y Huang - Molecular Therapy-Nucleic Acids, 2017 - cell.com
A 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 …
delivery of them into targeted tissues and cells. N-acetylgalactosamine (GalNAc) is a well …