Gene Therapy for Neurodegenerative Disease: Clinical Potential and Directions

被引：16

作者：

Zhu, Xiaolin ^{[1
]}

Zhang, Yu ^{[1
]}

Yang, Xin ^{[1
]}

Hao, Chunyan ^{[2
]}

Duan, Hubin ^{[1
,3
]}

机构：

[1] Shanxi Med Univ, Hosp 1, Dept Neurosurg, Taiyuan, Peoples R China

[2] Shanxi Med Univ, Hosp 1, Dept Geriatr, Taiyuan, Peoples R China

[3] Lvliang Peoples Hosp, Dept Neurosurg, Lvliang, Peoples R China

来源：

FRONTIERS IN MOLECULAR NEUROSCIENCE | 2021年 / 14卷

关键词：

gene editing; neurodegenerative disease; Alzheimer's disease; Parkinson's disease; Huntington's disease; amyotrophic lateral sclerosis; HEXANUCLEOTIDE REPEAT EXPANSION; I-SCE-I; HOMOLOGOUS RECOMBINATION; ALZHEIMERS-DISEASE; DNA-BINDING; MOTOR-NEURONS; IMMUNE-SYSTEM; BREAK REPAIR; MOUSE MODEL; A-BETA;

D O I：

10.3389/fnmol.2021.618171

中图分类号：

Q189 [神经科学];

学科分类号：

071006 ;

摘要：

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.

引用

页数：10

共 138 条

[1] Correction of amyotrophic lateral sclerosis related phenotypes in induced pluripotent stem cell-derived motor neurons carrying a hexanucleotide expansion mutation in C9orf72 by CRISPR/Cas9 genome editing using homology-directed repair [J].

Ababneh, Nidaa A. ;

Scaber, Jakub ;

Flynn, Rowan ;

Douglas, Andrew ;

Barbagallo, Paola ;

Candalija, Ana ;

Turner, Martin R. ;

Sims, David ;

Dafinca, Ruxandra ;

Cowley, Sally A. ;

Talbot, Kevin .

HUMAN MOLECULAR GENETICS, 2020, 29 (13) :2200-2217

[2] Knocking out C9ORF72 Exacerbates Axonal Trafficking Defects Associated with Hexanucleotide Repeat Expansion and Reduces Levels of Heat Shock Proteins [J].

Abo-Rady, Masin ;

Kalmbach, Norman ;

Pal, Arun ;

Schludi, Carina ;

Janosch, Antje ;

Richter, Tanja ;

Freitag, Petra ;

Bickle, Marc ;

Kahlert, Anne-Karin ;

Petri, Susanne ;

Stefanov, Stefan ;

Glass, Hannes ;

Staege, Selma ;

Just, Walter ;

Bhatnagar, Rajat ;

Edbauer, Dieter ;

Hermann, Andreas ;

Wegner, Florian ;

Sterneckert, Jared L. .

STEM CELL REPORTS, 2020, 14 (03) :390-405

[3] Pathogenic Pathways in Early-Onset Autosomal Recessive Parkinson's Disease Discovered Using Isogenic Human Dopaminergic Neurons [J].

Ahfeldt, Tim ;

Ordureau, Alban ;

Bell, Christina ;

Sarrafha, Lily ;

Sun, Chicheng ;

Piccinotti, Silvia ;

Grass, Tobias ;

Parfitt, Gustavo M. ;

Paulo, Joao A. ;

Yanagawa, Fumiki ;

Uozumi, Takayuki ;

Kiyota, Yasujiro ;

Harper, J. Wade ;

Rubin, Lee L. .

STEM CELL REPORTS, 2020, 14 (01) :75-90

[4] DNA-dependent protein kinase suppresses double-strand break-induced and spontaneous homologous recombination [J].

Allen, C ;

Kurimasa, A ;

Brenneman, MA ;

Chen, DJ ;

Nickoloff, JA .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2002, 99 (06) :3758-3763

[5]

An M.C., 2014, PLoS Curr, V6

[6] Genetic Correction of Huntington's Disease Phenotypes in Induced Pluripotent Stem Cells [J].

An, Mahru C. ;

Zhang, Ningzhe ;

Scott, Gary ;

Montoro, Daniel ;

Wittkop, Tobias ;

Mooney, Sean ;

Melov, Simon ;

Ellerby, Lisa M. .

CELL STEM CELL, 2012, 11 (02) :253-263

[7] Dipeptide repeat proteins inhibit homology-directed DNA double strand break repair in C9ORF72 ALS/FTD [J].

Andrade, Nadja S. ;

Ramic, Melina ;

Esanov, Rustam ;

Liu, Wenjun ;

Rybin, Mathew J. ;

Gaidosh, Gabriel ;

Abdallah, Abbas ;

Del'Olio, Samuel ;

Huff, Tyler C. ;

Chee, Nancy T. ;

Anatha, Sadhana ;

Gendron, Tania F. ;

Wahlestedt, Claes ;

Zhang, Yanbin ;

Benatar, Michael ;

Mueller, Christian ;

Zeier, Zane .

MOLECULAR NEURODEGENERATION, 2020, 15 (01)

[8] Search-and-replace genome editing without double-strand breaks or donor DNA [J].

Anzalone, Andrew V. ;

Randolph, Peyton B. ;

Davis, Jessie R. ;

Sousa, Alexander A. ;

Koblan, Luke W. ;

Levy, Jonathan M. ;

Chen, Peter J. ;

Wilson, Christopher ;

Newby, Gregory A. ;

Raguram, Aditya ;

Liu, David R. .

NATURE, 2019, 576 (7785) :149-+

[9] FACS-Assisted CRISPR-Cas9 Genome Editing Facilitates Parkinson's Disease Modeling [J].

Arias-Fuenzalida, Jonathan ;

Jarazo, Javier ;

Qing, Xiaobing ;

Walter, Jonas ;

Gomez-Giro, Gemma ;

Nickels, Sarah Louise ;

Zaehres, Holm ;

Schoeler, Hans Robert ;

Schwamborn, Jens Christian .

STEM CELL REPORTS, 2017, 9 (05) :1423-1431

[10] Sex and APOE ε4 genotype modify the Alzheimer's disease serum metabolome [J].

Arnold, Matthias ;

Nho, Kwangsik ;

Kueider-Paisley, Alexandra ;

Massaro, Tyler ;

Huynh, Kevin ;

Brauner, Barbara ;

MahmoudianDehkordi, Siamak ;

Louie, Gregory ;

Moseley, M. Arthur ;

Thompson, J. Will ;

St John-Williams, Lisa ;

Tenenbaum, Jessica D. ;

Blach, Colette ;

Chang, Rui ;

Brinton, Roberta D. ;

Baillie, Rebecca ;

Han, Xianlin ;

Trojanowski, John Q. ;

Shaw, Leslie M. ;

Martins, Ralph ;

Weiner, Michael W. ;

Trushina, Eugenia ;

Toledo, Jon B. ;

Meikle, Peter J. ;

Bennett, David A. ;

Krumsiek, Jan ;

Doraiswamy, P. Murali ;

Saykin, Andrew J. ;

Kaddurah-Daouk, Rima ;

Kastenmueller, Gabi .

NATURE COMMUNICATIONS, 2020, 11 (01)

← 1 2 3 4 5 6 7 8 9 10 →