Lipid nanoparticle technology for therapeutic gene regulation in the liver
Hereditary genetic disorders, cancer, and infectious diseases of the liver affect millions of
people around the globe and are a major public health burden. Most contemporary …
people around the globe and are a major public health burden. Most contemporary …
[HTML][HTML] Development of novel siRNA therapeutics: a review with a focus on inclisiran for the treatment of hypercholesterolemia
O Ebenezer, P Comoglio, GKS Wong… - International Journal of …, 2023 - mdpi.com
Over the past two decades, it was discovered that introducing synthetic small interfering
RNAs (siRNAs) into the cytoplasm facilitates effective gene-targeted silencing. This …
RNAs (siRNAs) into the cytoplasm facilitates effective gene-targeted silencing. This …
The nonclinical disposition and pharmacokinetic/pharmacodynamic properties of N-Acetylgalactosamine–conjugated small interfering RNA are highly predictable and …
Conjugation of oligonucleotide therapeutics, including small interfering RNAs (siRNAs) or
antisense oligonucleotides, to N-acetylgalactosamine (GalNAc) ligands has become the …
antisense oligonucleotides, to N-acetylgalactosamine (GalNAc) ligands has become the …
[HTML][HTML] Cancer nanotechnology: Enhancing tumor cell response to chemotherapy for hepatocellular carcinoma therapy
Hepatocellular carcinoma (HCC) is one of the deadliest cancers due to its complexities,
reoccurrence after surgical resection, metastasis and heterogeneity. In addition to sorafenib …
reoccurrence after surgical resection, metastasis and heterogeneity. In addition to sorafenib …
Recent progress of glycopolymer synthesis for biomedical applications
I Pramudya, H Chung - Biomaterials science, 2019 - pubs.rsc.org
Glycopolymers are an important class of biomaterials which include carbohydrate moieties
in their polymer structure. In addition to biological research on the interactions of …
in their polymer structure. In addition to biological research on the interactions of …
[HTML][HTML] 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 …
Pharmacokinetics and Clinical Pharmacology Considerations of GalNAc3-Conjugated Antisense Oligonucleotides
Y Wang, RZ Yu, S Henry, RS Geary - Expert opinion on drug …, 2019 - Taylor & Francis
Introduction: Triantennary N-acetyl galactosamine (GalNAc3)–conjugated antisense
oligonucleotides (ASOs) have demonstrated improved hepatocyte uptake and …
oligonucleotides (ASOs) have demonstrated improved hepatocyte uptake and …
Clinical Pharmacology of RNA Interference–Based Therapeutics: A Summary Based on Food and Drug Administration–Approved Small Interfering RNAs
RNA-based oligonucleotide therapeutics are revolutionizing drug development for disease
treatment. This class of therapeutics differs from small molecules and protein therapeutics in …
treatment. This class of therapeutics differs from small molecules and protein therapeutics in …
Development of T cells carrying two complementary chimeric antigen receptors against glypican-3 and asialoglycoprotein receptor 1 for the treatment of hepatocellular …
C Chen, K Li, H Jiang, F Song, H Gao, X Pan… - Cancer Immunology …, 2017 - Springer
Adoptive immunotherapy leveraging chimeric antigen receptor-modified T (CAR-T) cells
holds great promise for the treatment of cancer. However, tumor-associated antigens often …
holds great promise for the treatment of cancer. However, tumor-associated antigens often …
Inclisiran for the treatment of hypercholesterolaemia: implications and unanswered questions from the ORION trials
AJ Cupido, JJP Kastelein - Cardiovascular research, 2020 - academic.oup.com
Recently, three back-to-back published randomized clinical trials provided convincing
results for the novel lipid-lowering agent Inclisiran. Inclisiran is a small interfering RNA …
results for the novel lipid-lowering agent Inclisiran. Inclisiran is a small interfering RNA …