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Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB

The era of the aging society has arrived, and this is accompanied by an increase in the absolute numbers of patients with neurological disorders, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD). Such neurological disorders are serious costly diseases that have a significant impact on s...

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Autores principales: Kimura, Seigo, Harashima, Hideyoshi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7765480/
https://www.ncbi.nlm.nih.gov/pubmed/33334049
http://dx.doi.org/10.3390/pharmaceutics12121216
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author Kimura, Seigo
Harashima, Hideyoshi
author_facet Kimura, Seigo
Harashima, Hideyoshi
author_sort Kimura, Seigo
collection PubMed
description The era of the aging society has arrived, and this is accompanied by an increase in the absolute numbers of patients with neurological disorders, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD). Such neurological disorders are serious costly diseases that have a significant impact on society, both globally and socially. Gene therapy has great promise for the treatment of neurological disorders, but only a few gene therapy drugs are currently available. Delivery to the brain is the biggest hurdle in developing new drugs for the central nervous system (CNS) diseases and this is especially true in the case of gene delivery. Nanotechnologies such as viral and non-viral vectors allow efficient brain-targeted gene delivery systems to be created. The purpose of this review is to provide a comprehensive review of the current status of the development of successful drug delivery to the CNS for the treatment of CNS-related disorders especially by gene therapy. We mainly address three aspects of this situation: (1) blood-brain barrier (BBB) functions; (2) adeno-associated viral (AAV) vectors, currently the most advanced gene delivery vector; (3) non-viral brain targeting by non-invasive methods.
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spelling pubmed-77654802020-12-27 Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB Kimura, Seigo Harashima, Hideyoshi Pharmaceutics Review The era of the aging society has arrived, and this is accompanied by an increase in the absolute numbers of patients with neurological disorders, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD). Such neurological disorders are serious costly diseases that have a significant impact on society, both globally and socially. Gene therapy has great promise for the treatment of neurological disorders, but only a few gene therapy drugs are currently available. Delivery to the brain is the biggest hurdle in developing new drugs for the central nervous system (CNS) diseases and this is especially true in the case of gene delivery. Nanotechnologies such as viral and non-viral vectors allow efficient brain-targeted gene delivery systems to be created. The purpose of this review is to provide a comprehensive review of the current status of the development of successful drug delivery to the CNS for the treatment of CNS-related disorders especially by gene therapy. We mainly address three aspects of this situation: (1) blood-brain barrier (BBB) functions; (2) adeno-associated viral (AAV) vectors, currently the most advanced gene delivery vector; (3) non-viral brain targeting by non-invasive methods. MDPI 2020-12-15 /pmc/articles/PMC7765480/ /pubmed/33334049 http://dx.doi.org/10.3390/pharmaceutics12121216 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Kimura, Seigo
Harashima, Hideyoshi
Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
title Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
title_full Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
title_fullStr Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
title_full_unstemmed Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
title_short Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
title_sort current status and challenges associated with cns-targeted gene delivery across the bbb
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7765480/
https://www.ncbi.nlm.nih.gov/pubmed/33334049
http://dx.doi.org/10.3390/pharmaceutics12121216
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