CRISPR-Cas9-Mediated NPC1 Gene Deletion Enhances HEK 293 T Cell Adhesion by Regulating E-Cadherin DOI

Zisen Jia,

Minlin Yang,

Yanchun Zhao

et al.

Molecular Biotechnology, Journal Year: 2022, Volume and Issue: 65(2), P. 252 - 262

Published: May 19, 2022

Language: Английский

Current and promising therapeutic options for Dravet syndrome DOI
Antonella Riva, Gianluca D’Onofrio, Elisabetta Amadori

et al.

Expert Opinion on Pharmacotherapy, Journal Year: 2022, Volume and Issue: 23(15), P. 1727 - 1736

Published: Sept. 20, 2022

Dravet Syndrome (DS) is a developmental and epileptic encephalopathy carrying high-level psychobehavioral burdens. Although the disease has been known for almost 4 decades, despite significant progress in understanding of its physiopathology natural course, pharmacological treatment leaves patients caregivers with unmet needs. This review provides summary current promising therapeutic options DS.PubMed ClinicalTrials.gov were screened using 'Dravet Syndrome' OR 'DS,' AND 'pharmacotherapy,' 'treatments.' Randomized clinical trials, structured reviews, meta-analyses selected in-human application well-known anti-seizure medications; while in-vivo experiments on models DS to evaluate potential new strategies.The search led by need care defeat course disease, an aspect still largely neglected available strategies. Yet, last 6 years have climate increased interest availability trials. Particularly, gene therapy could hopefully prevent evolution directly relieving specific genetic defect, although possibility off-target editing, uneasy administration route prevented use.

Language: Английский

Citations

9
GEARBOCS: An Adeno Associated Virus Tool forIn VivoGene Editing in Astrocytes DOI Creative Commons

Dhanesh Sivadasan Bindu,

Justin T Savage, Nicholas Brose

et al.

bioRxiv (Cold Spring Harbor Laboratory), Journal Year: 2023, Volume and Issue: unknown

Published: Jan. 19, 2023

Summary CRISPR/Cas9-based genome engineering enables rapid and precise gene manipulations in the CNS. Here, we developed a non-invasive astrocyte-specific method utilizing single AAV vector, which named GEARBOCS (Gene Editing AstRocytes Based On CRISPR/Cas9 System). We verified GEARBOCS’ specificity to mouse cortical astrocytes demonstrated its utility for three types of manipulations: knockout (KO); tagging (TagIn); reporter knock-in (GeneTrap) strategies. Next, deployed two test cases. First, determined that are necessary source synaptogenic factor Sparcl1 thalamocortical synapse maintenance primary visual cortex. Second, express synaptic vesicle associated Vamp2 protein found it is required maintaining excitatory inhibitory numbers These results show strategy provides fast efficient means study astrocyte biology vivo . Motivation Astrocytes indispensable brain development, function, health. However, molecular tools function have been largely limited genetically modified mice. editing within vector astrocytes. designed optimized this easy-to-use viral tool understand expression, localization

Language: Английский

Citations

4
CRISPR/Cas-mediated genome editing in mice for the development of drug delivery mechanism DOI

Sowbhagya Ramachandregowda,

Harsha Muktha,

Thippenahalli Narasimhaiah Ramakrishnaiah

et al.

Molecular Biology Reports, Journal Year: 2023, Volume and Issue: 50(9), P. 7729 - 7743

Published: July 12, 2023

Language: Английский

Citations

4
CRISPR-Cpf1 system and its applications in animal genome editing DOI

Yawei Han,

Zisen Jia,

Keli Xu

et al.

Molecular Genetics and Genomics, Journal Year: 2024, Volume and Issue: 299(1)

Published: Aug. 1, 2024

Language: Английский

Citations

1
CRISPR-Cas9-Mediated NPC1 Gene Deletion Enhances HEK 293 T Cell Adhesion by Regulating E-Cadherin DOI

Zisen Jia,

Minlin Yang,

Yanchun Zhao

et al.

Molecular Biotechnology, Journal Year: 2022, Volume and Issue: 65(2), P. 252 - 262

Published: May 19, 2022

Language: Английский

Citations

5