Vector enabled CRISPR gene editing – A revolutionary strategy for targeting the diversity of brain pathologies

Forgham H.; Liu L.; Zhu J.Javed I.Qiao R.Davis T.P.Cai W.

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Vector enabled CRISPR gene editing – A revolutionary strategy for targeting the diversity of brain pathologies

机译：Vector enabled CRISPR gene editing – A revolutionary strategy for targeting the diversity of brain pathologies

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? 2023 Elsevier B.V.Brain pathologies are considered one of the greatest contributors of death and disability worldwide. Neurodegenerative Alzheimer's disease is the second leading cause of death in adults, whilst brain cancers including glioblastoma multiforme in adults, and pediatric-type high-grade gliomas in children remain largely untreatable. A further compounding issue for patients with brain pathologies is that of long-term neuropsychiatric sequela - as a symptom or arising from high dose therapeutic intervention. The major challenge to effective, low dose treatment is finding therapeutics that successfully cross the blood–brain barrier and target aberrant cellular processes, while having minimum effect on essential cellular processes, and healthy bystander cells. Following over 30 years of research, CRISPR technology has emerged as a biomedical tour de force with the potential to revolutionise the treatment of both neurological and cancer related brain pathologies. The aim of this review is to take stock of the progress made in CRISPR technology in relation to treating brain pathologies. Specifically, we will describe studies which look beyond design, synthesis, and theoretical application; and focus instead on in vivo studies with translation potential. Along with discussing the latest breakthrough techniques being applied within the CRISPR field, we aim to provide a prospective on the knowledge gaps that exist and challenges that still lay ahead for CRISPR technology prior to successful application in the brain disease treatment field.

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《Coordination chemistry reviews》 |2023年第7期|215172.1-215172.22|共22页
作者
Forgham H.; Liu L.; Zhu J.Javed I.Qiao R.Davis T.P.Cai W.;
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作者单位

Australian Institute of Bioengineering & Nanotechnology The University of Queensland;

Departments of Radiology and Medical Physics University of Wisconsin – Madison;

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收录信息美国《科学引文索引》(SCI);美国《生物学医学文摘》(MEDLINE);
原文格式 PDF
正文语种英语
中图分类无机化学;
关键词
Blood–brain barrier (BBB); Brain; Cancer; Central nervous system (CNS); Clustered regularly interspaced short palindromic repeats (CRISPR); Gene editing;

Vector enabled CRISPR gene editing – A revolutionary strategy for targeting the diversity of brain pathologies

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