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

Poojitha Pinjala; Kamatham Pushpa Tryphena; Renuka Prasad; Dharmendra Kumar Khatri; Woong Sun; Shashi Bala Singh; Dalapathi Gugulothu; Saurabh Srivastava; Lalitkumar Vora

doi:10.1186/s40824-023-00381-y

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

Poojitha Pinjala, Kamatham Pushpa Tryphena, Renuka Prasad, Dharmendra Kumar Khatri^*, Woong Sun, Shashi Bala Singh, Dalapathi Gugulothu, Saurabh Srivastava, Lalitkumar Vora^*

^*Corresponding author for this work

School of Pharmacy

Research output: Contribution to journal › Review article › peer-review

22 Citations (Scopus)

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Abstract

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.

Original language	English
Article number	46
Number of pages	18
Journal	Biomaterials Research
Volume	27
DOIs	https://doi.org/10.1186/s40824-023-00381-y
Publication status	Published - 16 May 2023

Keywords

Biomedical Engineering
Medicine (miscellaneous)
Ceramics and Composites
Biomaterials

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10.1186/s40824-023-00381-yLicence: CC BY

CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease
Copyright 2023 The Authors. This is an open access article published under a Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium, provided the author and source are cited.
Final published version, 4.37 MBLicence: CC BY

Cite this

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title = "CRISPR/Cas9 assisted stem cell therapy in Parkinson's disease",

abstract = "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.",

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author = "Poojitha Pinjala and Tryphena, {Kamatham Pushpa} and Renuka Prasad and Khatri, {Dharmendra Kumar} and Woong Sun and Singh, {Shashi Bala} and Dalapathi Gugulothu and Saurabh Srivastava and Lalitkumar Vora",

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AU - Pinjala, Poojitha

AU - Tryphena, Kamatham Pushpa

AU - Prasad, Renuka

AU - Khatri, Dharmendra Kumar

AU - Sun, Woong

AU - Singh, Shashi Bala

AU - Gugulothu, Dalapathi

AU - Srivastava, Saurabh

AU - Vora, Lalitkumar

PY - 2023/5/16

Y1 - 2023/5/16

N2 - 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.

AB - 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.

KW - Biomedical Engineering

KW - Medicine (miscellaneous)

KW - Ceramics and Composites

KW - Biomaterials

U2 - 10.1186/s40824-023-00381-y

DO - 10.1186/s40824-023-00381-y

M3 - Review article

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SN - 2055-7124

VL - 27

JO - Biomaterials Research

JF - Biomaterials Research

M1 - 46

ER -

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

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Keywords

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