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CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease

Since its discovery in 2012, CRISPR Cas9 has been tried as a direct treatment approach to correct the causative gene mutation and establish animal models in neurodegenerative disorders. Since no strategy developed until now could completely cure Parkinson's disease (PD), neuroscientists aspire...

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Autores principales: Pinjala, Poojitha, Tryphena, Kamatham Pushpa, Prasad, Renuka, Khatri, Dharmendra Kumar, Sun, Woong, Singh, Shashi Bala, Gugulothu, Dalapathi, Srivastava, Saurabh, Vora, Lalitkumar
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10190035/
https://www.ncbi.nlm.nih.gov/pubmed/37194005
http://dx.doi.org/10.1186/s40824-023-00381-y
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author Pinjala, Poojitha
Tryphena, Kamatham Pushpa
Prasad, Renuka
Khatri, Dharmendra Kumar
Sun, Woong
Singh, Shashi Bala
Gugulothu, Dalapathi
Srivastava, Saurabh
Vora, Lalitkumar
author_facet Pinjala, Poojitha
Tryphena, Kamatham Pushpa
Prasad, Renuka
Khatri, Dharmendra Kumar
Sun, Woong
Singh, Shashi Bala
Gugulothu, Dalapathi
Srivastava, Saurabh
Vora, Lalitkumar
author_sort Pinjala, Poojitha
collection PubMed
description Since its discovery in 2012, CRISPR Cas9 has been tried as a direct treatment approach to correct the causative gene mutation and establish animal models in neurodegenerative disorders. Since no strategy developed until now could completely cure Parkinson's disease (PD), neuroscientists aspire to use gene editing technology, especially CRISPR/Cas9, to induce a permanent correction in genetic PD patients expressing mutated genes. Over the years, our understanding of stem cell biology has improved. Scientists have developed personalized cell therapy using CRISPR/Cas9 to edit embryonic and patient-derived stem cells ex-vivo. This review details the importance of CRISPR/Cas9-based stem cell therapy in Parkinson's disease in developing PD disease models and developing therapeutic strategies after elucidating the possible pathophysiological mechanisms. GRAPHICAL ABSTRACT: [Image: see text]
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spelling pubmed-101900352023-05-18 CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease Pinjala, Poojitha Tryphena, Kamatham Pushpa Prasad, Renuka Khatri, Dharmendra Kumar Sun, Woong Singh, Shashi Bala Gugulothu, Dalapathi Srivastava, Saurabh Vora, Lalitkumar Biomater Res Review Since its discovery in 2012, CRISPR Cas9 has been tried as a direct treatment approach to correct the causative gene mutation and establish animal models in neurodegenerative disorders. Since no strategy developed until now could completely cure Parkinson's disease (PD), neuroscientists aspire to use gene editing technology, especially CRISPR/Cas9, to induce a permanent correction in genetic PD patients expressing mutated genes. Over the years, our understanding of stem cell biology has improved. Scientists have developed personalized cell therapy using CRISPR/Cas9 to edit embryonic and patient-derived stem cells ex-vivo. This review details the importance of CRISPR/Cas9-based stem cell therapy in Parkinson's disease in developing PD disease models and developing therapeutic strategies after elucidating the possible pathophysiological mechanisms. GRAPHICAL ABSTRACT: [Image: see text] BioMed Central 2023-05-16 /pmc/articles/PMC10190035/ /pubmed/37194005 http://dx.doi.org/10.1186/s40824-023-00381-y Text en © The Author(s) 2023 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) . The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/ (https://creativecommons.org/publicdomain/zero/1.0/) ) applies to the data made available in this article, unless otherwise stated in a credit line to the data.
spellingShingle Review
Pinjala, Poojitha
Tryphena, Kamatham Pushpa
Prasad, Renuka
Khatri, Dharmendra Kumar
Sun, Woong
Singh, Shashi Bala
Gugulothu, Dalapathi
Srivastava, Saurabh
Vora, Lalitkumar
CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
title CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
title_full CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
title_fullStr CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
title_full_unstemmed CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
title_short CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
title_sort crispr/cas9 assisted stem cell therapy in parkinson's disease
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10190035/
https://www.ncbi.nlm.nih.gov/pubmed/37194005
http://dx.doi.org/10.1186/s40824-023-00381-y
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