Advances in Gene Therapy Techniques to Treat Gene Mutation

Overview

Journal Biomolecules

Publisher MDPI

Specialties Biochemistry
Molecular Biology

Date 2022 Dec 23

PMID 36551242

Authors

Sun-Ku Chung

Seo-Young Lee

Affiliations

Soon will be listed here.

Abstract

Leucine-rich repeat kinase 2 () gene mutation is an autosomal dominant mutation associated with Parkinson's disease (PD). Among gene mutations, the G2019S mutation is frequently involved in PD onset. Currently, diverse gene correction tools such as zinc finger nucleases (ZFNs), helper-dependent adenoviral vector (HDAdV), the bacterial artificial chromosome-based homologous recombination (BAC-based HR) system, and CRISPR/Cas9-homology-directed repair (HDR) or adenine base editor (ABE) are used in genome editing. Gene correction of the G2019S mutation has been applied whenever new gene therapy tools emerge, being mainly applied to induced pluripotent stem cells ( G2019S-mutant iPSCs). Here, we comprehensively introduce the principles and methods of each programmable nuclease such as ZFN, CRISPR/Cas9-HDR or ABE applied to G2019S, as well as those of HDAdV or BAC-based HR systems used as nonprogrammable nuclease systems.

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