TY - JOUR
T1 - CRISPR/Cas9 gene editing
T2 - New hope for Alzheimer's disease therapeutics
AU - Bhardwaj, Shanu
AU - Kesari, Kavindra Kumar
AU - Rachamalla, Mahesh
AU - Mani, Shalini
AU - Ashraf, Ghulam Md
AU - Jha, Saurabh Kumar
AU - Kumar, Pravir
AU - Ambasta, Rashmi K.
AU - Dureja, Harish
AU - Devkota, Hari Prasad
AU - Gupta, Gaurav
AU - Chellappan, Dinesh Kumar
AU - Singh, Sachin Kumar
AU - Dua, Kamal
AU - Ruokolainen, Janne
AU - Kamal, Mohammad Amjad
AU - Ojha, Shreesh
AU - Jha, Niraj Kumar
N1 - Publisher Copyright:
© 2021
PY - 2022/9
Y1 - 2022/9
N2 - Background: Alzheimer's disease (AD) is an insidious, irreversible, and progressive neurodegenerative health condition manifesting as cognitive deficits and amyloid beta (Aβ) plaques and neurofibrillary tangles. Approximately 50 million individuals are affected by AD, and the number is rapidly increasing globally. This review explores the role of CRISPR/Cas9 gene editing in the management of AD and its clinical manifestations. Aim of Review: This review aims to provide a deep insight into the recent progress in CRISPR/Cas9-mediated genome editing and its use against neurodegenerative disorders, specifically AD. However, we have referred to its use against parkinsons's disease (PD), Huntington's disease (HD), and other human diseases, as is one of the most promising and emerging technologies for disease treatment. Key Scientific Concepts of Review: The pathophysiology of AD is known to be linked with gene mutations, that is, presenilin (PSEN) and amyloid beta precursor protein (APP). However, clinical trials focused at the genetic level could not meet the desired efficiency. The CRISPR/Cas9 genome editing tool is one of the most powerful technologies for correcting inconsistent genetic signatures and now extensively used for AD management. It has significant potential for the correction of undesired gene mutations associated with AD. This technology has allowed the development of empirical AD models, therapeutic lines, and diagnostic approaches for better understanding the nervous system, from in vitro to in vivo models.
AB - Background: Alzheimer's disease (AD) is an insidious, irreversible, and progressive neurodegenerative health condition manifesting as cognitive deficits and amyloid beta (Aβ) plaques and neurofibrillary tangles. Approximately 50 million individuals are affected by AD, and the number is rapidly increasing globally. This review explores the role of CRISPR/Cas9 gene editing in the management of AD and its clinical manifestations. Aim of Review: This review aims to provide a deep insight into the recent progress in CRISPR/Cas9-mediated genome editing and its use against neurodegenerative disorders, specifically AD. However, we have referred to its use against parkinsons's disease (PD), Huntington's disease (HD), and other human diseases, as is one of the most promising and emerging technologies for disease treatment. Key Scientific Concepts of Review: The pathophysiology of AD is known to be linked with gene mutations, that is, presenilin (PSEN) and amyloid beta precursor protein (APP). However, clinical trials focused at the genetic level could not meet the desired efficiency. The CRISPR/Cas9 genome editing tool is one of the most powerful technologies for correcting inconsistent genetic signatures and now extensively used for AD management. It has significant potential for the correction of undesired gene mutations associated with AD. This technology has allowed the development of empirical AD models, therapeutic lines, and diagnostic approaches for better understanding the nervous system, from in vitro to in vivo models.
KW - Alzheimer's disease
KW - APP
KW - CRISPR/Cas9
KW - Gene editing
KW - Neurodegeneration
KW - Presenilin
KW - Therapeutics
UR - http://www.scopus.com/inward/record.url?scp=85111514756&partnerID=8YFLogxK
U2 - 10.1016/j.jare.2021.07.001
DO - 10.1016/j.jare.2021.07.001
M3 - Article
AN - SCOPUS:85111514756
SN - 2090-1232
VL - 40
SP - 207
EP - 221
JO - Journal of Advanced Research
JF - Journal of Advanced Research
ER -