Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions

The pathogenesis of neurodegenerative diseases (NDDs) is complex and diverse. Over the decades, our understanding of NDD has been limited to pathological features. However, recent advances in gene sequencing have facilitated elucidation of NDD at a deeper level. Gene editing techniques have uncovere...

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Main Authors: Xiaolin Zhu, Yu Zhang, Xin Yang, Chunyan Hao, Hubin Duan
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-06-01
Series:Frontiers in Molecular Neuroscience
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fnmol.2021.618171/full
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author Xiaolin Zhu
Yu Zhang
Xin Yang
Chunyan Hao
Hubin Duan
Hubin Duan
author_facet Xiaolin Zhu
Yu Zhang
Xin Yang
Chunyan Hao
Hubin Duan
Hubin Duan
author_sort Xiaolin Zhu
collection DOAJ
description The pathogenesis of neurodegenerative diseases (NDDs) is complex and diverse. Over the decades, our understanding of NDD has been limited to pathological features. However, recent advances in gene sequencing have facilitated elucidation of NDD at a deeper level. Gene editing techniques have uncovered new genetic links to phenotypes, promoted the development of novel treatment strategies and equipped researchers with further means to construct effective cell and animal models. The current review describes the history of evolution of gene editing tools, with the aim of improving overall understanding of this technology, and focuses on the four most common NDD disorders to demonstrate the potential future applications and research directions of gene editing.
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spelling doaj.art-d564a11c3a234689bab16ac967656ab32022-12-21T21:59:48ZengFrontiers Media S.A.Frontiers in Molecular Neuroscience1662-50992021-06-011410.3389/fnmol.2021.618171618171Gene Therapy for Neurodegenerative Disease: Clinical Potential and DirectionsXiaolin Zhu0Yu Zhang1Xin Yang2Chunyan Hao3Hubin Duan4Hubin Duan5Department of Neurosurgery, First Hospital of Shanxi Medical University, Taiyuan, ChinaDepartment of Neurosurgery, First Hospital of Shanxi Medical University, Taiyuan, ChinaDepartment of Neurosurgery, First Hospital of Shanxi Medical University, Taiyuan, ChinaDepartment of Geriatrics, First Hospital of Shanxi Medical University, Taiyuan, ChinaDepartment of Neurosurgery, First Hospital of Shanxi Medical University, Taiyuan, ChinaDepartment of Neurosurgery, Lvliang People’s Hospital, Lvliang, ChinaThe pathogenesis of neurodegenerative diseases (NDDs) is complex and diverse. Over the decades, our understanding of NDD has been limited to pathological features. However, recent advances in gene sequencing have facilitated elucidation of NDD at a deeper level. Gene editing techniques have uncovered new genetic links to phenotypes, promoted the development of novel treatment strategies and equipped researchers with further means to construct effective cell and animal models. The current review describes the history of evolution of gene editing tools, with the aim of improving overall understanding of this technology, and focuses on the four most common NDD disorders to demonstrate the potential future applications and research directions of gene editing.https://www.frontiersin.org/articles/10.3389/fnmol.2021.618171/fullgene editingneurodegenerative diseaseAlzheimer’s diseaseParkinson’s diseaseHuntington’s diseaseamyotrophic lateral sclerosis
spellingShingle Xiaolin Zhu
Yu Zhang
Xin Yang
Chunyan Hao
Hubin Duan
Hubin Duan
Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions
Frontiers in Molecular Neuroscience
gene editing
neurodegenerative disease
Alzheimer’s disease
Parkinson’s disease
Huntington’s disease
amyotrophic lateral sclerosis
title Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions
title_full Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions
title_fullStr Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions
title_full_unstemmed Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions
title_short Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions
title_sort gene therapy for neurodegenerative disease clinical potential and directions
topic gene editing
neurodegenerative disease
Alzheimer’s disease
Parkinson’s disease
Huntington’s disease
amyotrophic lateral sclerosis
url https://www.frontiersin.org/articles/10.3389/fnmol.2021.618171/full
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AT yuzhang genetherapyforneurodegenerativediseaseclinicalpotentialanddirections
AT xinyang genetherapyforneurodegenerativediseaseclinicalpotentialanddirections
AT chunyanhao genetherapyforneurodegenerativediseaseclinicalpotentialanddirections
AT hubinduan genetherapyforneurodegenerativediseaseclinicalpotentialanddirections
AT hubinduan genetherapyforneurodegenerativediseaseclinicalpotentialanddirections