Lipid nanoparticles for nucleic acid delivery to endothelial cells

GW Liu, EB Guzman, N Menon, RS Langer - Pharmaceutical Research, 2023 - Springer
Endothelial cells play critical roles in circulatory homeostasis and are also the gateway to
the major organs of the body. Dysfunction, injury, and gene expression profiles of these cells …

Emerging nanobiotechnology for precise theranostics of hepatocellular carcinoma

M Xu, L Yang, Y Lin, Y Lu, X Bi, T Jiang… - Journal of …, 2022 - Springer
Primary liver cancer has become the second most fatal cancer in the world, and its five-year
survival rate is only 10%. Most patients are in the middle and advanced stages at the time of …

Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

SQ Liang, P Liu, JL Smith, E Mintzer, S Maitland… - Nature …, 2022 - nature.com
Abstract Analysis of off-target editing is an important aspect of the development of safe
nuclease-based genome editing therapeutics. in vivo assessment of nuclease off-target …

[HTML][HTML] Research progress and application of the CRISPR/Cas9 gene-editing technology based on hepatocellular carcinoma

S Yu, R Zhao, B Zhang, C Lai, L Li, J Shen… - Asian Journal of …, 2023 - Elsevier
Hepatocellular carcinoma (HCC) is now a common cause of cancer death, with no obvious
change in patient survival over the past few years. Although the traditional therapeutic …

[HTML][HTML] Using CRISPR/Cas9 to model human liver disease

M Alves-Bezerra, N Furey, CG Johnson, KD Bissig - JHEP Reports, 2019 - Elsevier
CRISPR/Cas9 gene editing has revolutionised biomedical research. The ease of design has
allowed many groups to apply this technology for disease modelling in animals. While the …

SEMMs: Somatically Engineered Mouse Models. A New Tool for In Vivo Disease Modeling for Basic and Translational Research

A Lima, D Maddalo - Frontiers in Oncology, 2021 - frontiersin.org
Most experimental oncology therapies fail during clinical development despite years of
preclinical testing rationalizing their use. This begs the question of whether the current …

[HTML][HTML] Selecting an appropriate experimental animal model for cholangiocarcinoma research

M Li, X Zhou, W Wang, B Ji, Y Shao, Q Du… - Journal of Clinical …, 2022 - ncbi.nlm.nih.gov
Cholangiocarcinoma (CCA) is a highly aggressive biliary tree malignancy with intrahepatic
and extra-hepatic subtypes that differ in molecular pathogeneses, epidemiology, clinical …

CRISPR‐induced exon skipping of β‐catenin reveals tumorigenic mutants driving distinct subtypes of liver cancer

H Mou, O Eskiocak, KA Özler, M Gorman… - The Journal of …, 2023 - Wiley Online Library
CRISPR/Cas9‐driven cancer modeling studies are based on the disruption of tumor
suppressor genes by small insertions or deletions (indels) that lead to frame‐shift mutations …

[HTML][HTML] A spliceosome-associated gene signature aids in predicting prognosis and tumor microenvironment of hepatocellular carcinoma

H Wang, R Wang, J Fang - Aging (Albany NY), 2023 - ncbi.nlm.nih.gov
Splicing alterations have been shown to be key tumorigenesis drivers. In this study, we
identified a novel spliceosome-related genes (SRGs) signature to predict the overall survival …

Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy

ML Zhang, HB Li, Y Jin - Frontiers in Genetics, 2024 - frontiersin.org
The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) mediated Cas9
nuclease system has been extensively used for genome editing and gene modification in …