Cargando…

CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease

Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over...

Descripción completa

Detalles Bibliográficos
Autores principales: Rahman, Mujeeb ur, Bilal, Muhammad, Shah, Junaid Ali, Kaushik, Ajeet, Teissedre, Pierre-Louis, Kujawska, Małgorzata
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9229276/
https://www.ncbi.nlm.nih.gov/pubmed/35745824
http://dx.doi.org/10.3390/pharmaceutics14061252
_version_ 1784734702435827712
author Rahman, Mujeeb ur
Bilal, Muhammad
Shah, Junaid Ali
Kaushik, Ajeet
Teissedre, Pierre-Louis
Kujawska, Małgorzata
author_facet Rahman, Mujeeb ur
Bilal, Muhammad
Shah, Junaid Ali
Kaushik, Ajeet
Teissedre, Pierre-Louis
Kujawska, Małgorzata
author_sort Rahman, Mujeeb ur
collection PubMed
description Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques hold great promise for research and therapy of NDs, such as PD, for which there are currently no effective disease-modifying treatments. As a result, gene therapy may offer new treatment options, transforming our ability to treat this disease. We present a detailed overview of novel gene-editing delivery vehicles, which is essential for their successful implementation in both cutting-edge research and prospective therapeutics. Moreover, we review the most recent advancements in CRISPR-based applications and gene therapies for a better understanding of treating PD. We explore the benefits and drawbacks of using them for a range of gene-editing applications in the brain, emphasizing some fascinating possibilities.
format Online
Article
Text
id pubmed-9229276
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher MDPI
record_format MEDLINE/PubMed
spelling pubmed-92292762022-06-25 CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease Rahman, Mujeeb ur Bilal, Muhammad Shah, Junaid Ali Kaushik, Ajeet Teissedre, Pierre-Louis Kujawska, Małgorzata Pharmaceutics Review Parkinson’s disease (PD) and other chronic and debilitating neurodegenerative diseases (NDs) impose a substantial medical, emotional, and financial burden on individuals and society. The origin of PD is unknown due to a complex combination of hereditary and environmental risk factors. However, over the last several decades, a significant amount of available data from clinical and experimental studies has implicated neuroinflammation, oxidative stress, dysregulated protein degradation, and mitochondrial dysfunction as the primary causes of PD neurodegeneration. The new gene-editing techniques hold great promise for research and therapy of NDs, such as PD, for which there are currently no effective disease-modifying treatments. As a result, gene therapy may offer new treatment options, transforming our ability to treat this disease. We present a detailed overview of novel gene-editing delivery vehicles, which is essential for their successful implementation in both cutting-edge research and prospective therapeutics. Moreover, we review the most recent advancements in CRISPR-based applications and gene therapies for a better understanding of treating PD. We explore the benefits and drawbacks of using them for a range of gene-editing applications in the brain, emphasizing some fascinating possibilities. MDPI 2022-06-13 /pmc/articles/PMC9229276/ /pubmed/35745824 http://dx.doi.org/10.3390/pharmaceutics14061252 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/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 (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Rahman, Mujeeb ur
Bilal, Muhammad
Shah, Junaid Ali
Kaushik, Ajeet
Teissedre, Pierre-Louis
Kujawska, Małgorzata
CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
title CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
title_full CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
title_fullStr CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
title_full_unstemmed CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
title_short CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson’s Disease
title_sort crispr-cas9-based technology and its relevance to gene editing in parkinson’s disease
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9229276/
https://www.ncbi.nlm.nih.gov/pubmed/35745824
http://dx.doi.org/10.3390/pharmaceutics14061252
work_keys_str_mv AT rahmanmujeebur crisprcas9basedtechnologyanditsrelevancetogeneeditinginparkinsonsdisease
AT bilalmuhammad crisprcas9basedtechnologyanditsrelevancetogeneeditinginparkinsonsdisease
AT shahjunaidali crisprcas9basedtechnologyanditsrelevancetogeneeditinginparkinsonsdisease
AT kaushikajeet crisprcas9basedtechnologyanditsrelevancetogeneeditinginparkinsonsdisease
AT teissedrepierrelouis crisprcas9basedtechnologyanditsrelevancetogeneeditinginparkinsonsdisease
AT kujawskamałgorzata crisprcas9basedtechnologyanditsrelevancetogeneeditinginparkinsonsdisease