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 - Funding Information:
Dr. Shalini Mani is Associate Professor in the Department of Biotechnology at Jaypee Institute of Information Technology, Noida, India. She has total 17 years of research and 10 years of teaching experience. She completed her PhD from Centre for Cellular and Molecular Biology, Hyderabad, where she worked on detailed analysis of clinical biochemical and genetic analysis of Leigh syndrome patients. She also received a prestigious Endeavour Research fellowship award from Australia and did her post-doctorate from Murdoch Children Research Institute, Melbourne Australia. A resurgence of interest in the study of mitochondria during the past decades has revealed that mitochondria also play key roles in cell signaling, proliferation, cell metabolism and cell death, and that genetic and/or metabolic alterations in mitochondria contribute to a number of diseases, including cancer, diabetes and neurological disorders. Thus, as a part of fundamental research, her research interest primarily focuses on studying the role of mitochondria in different chronic diseases as well as effect of different environmental factors on mitochondrial metabolism. As a translational researcher, she is also exploring the molecular mechanism for therapeutic benefits of some selected herbs on mitochondrial activity in different diseases conditions such as diabetes, cancer and neurological disorders.
Funding Information:
Dr. Hari Prasad Devkota completed his Bachelor of Pharmaceutical Sciences (B. Pharm.) from Pokhara University, Nepal in 2005. After working at the same institute as Teaching Associate till 2007, he received Japanese Government (MEXT) Scholarship and entered Graduate School of Pharmaceutical Sciences, Kumamoto University, Japan in 2007 and completed Master and PhD in Pharmaceutical Sciences in 2010 and 2013, respectively. He was working as Postdoctoral Fellow in Kumamoto University (2013-2014) with the support from Takeda Science Foundation (TSF), Japan. From 2014, he is working as Assistant Professor at HIGO Program, Kumamoto University. His main research interests are plant-based bioactive natural products, functional foods and ethnopharmacology. Till now, he has authored more than 130 publications including original research articles, review articles, books and book chapters. He is actively involved in the research on medicinal plants from Asia and Africa for their ethnopharmacological surveys, chemical constituents and pharmacological activities.
Funding Information:
Dr. Sachin Kumar Singh is a Pharmaceutical Analyst and holds Ph.D degree in Pharmaceutical Sciences from Karpagam University, Coimbatore, India. Currently he is working as Professor, School of Pharmaceutical Sciences, Lovely Professional University (LPU), India. He has completed four industrial projects. Dr. Singh’s research work on polypeptide-k has got recognition from Punjab State Council for Science and Technology and DST/SERB. He has received a grant of 2.43 million under FastTrack Young Scientists Scheme for the development of SNEDDS of polypeptide-K for the effective treatment of diabetes mellitus. He has published more than 120 research papers in various national and international journals with cumulative impact factor 250 and h-index 21, filed ten patents and 10 international book chapters. He has supervised 30 M. Pharm projects and 8 PhD thesis and currently guiding 3 M. Pharm projects and 6 PhD thesis. He is reviewer of more than 40 international journals indexed in Web of Science and Scopus. Dr. Singh is a recipient of young scientist award, 2013 (DST/SERB govt of India), Bharat Shiksha Ratan Award, 2014 (G.S.H.E.G., New Delhi, India) and Dr. B. C. Deb Memorial Award, 2020 for popularization of science (Indian Science Congress Association, DST, India).
Funding Information:
Dr. Niraj Kumar Jha is currently Assistant Professor in the Department of Biotechnology, Sharda University, Greater Noida, India. He is a former Assistant Professor of the Faculty of Biotechnology, Noida Institute of Engineering and Technology (NIET), affiliated with Abdul Kalam Technical University (AKTU), India. He holds a Doctorate in Biotechnology from Delhi Technological University and a postgraduate degree in Biotechnology from Vellore Institute of Technology, TN, India. During the doctorate program, DBT granted him a fellowship as a financial boost to accomplish research work. He has published over 60 quality scholarly work and 5 book chapters in prestigious international journals and presented over 10 research papers in top national and international conferences. He is an editorial board member of various high repute international journals and assisted significantly in reviewing articles of many international journals such as Scientific reports, Life sciences, European journal of pharmaceutical sciences and Journal of food biochemistry. Currently, he is also serving as a guest editor for various top journals of international repute including Antioxidants, Environmental science and pollution research and Immuno. He has executed various research projects and supervised several doctoral scholars, masters, and bachelor students. His vast research expertise covers the areas of Natural products, Cell signaling and Disease.
Funding Information:
We are grateful to the United Arab Emirates University, UAE for the research grant support and also express gratitude to the unknown referees for their time and valuable suggestions to improve the manuscript.
Publisher Copyright:
© 2022
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 - APP
KW - Alzheimer's disease
KW - CRISPR/Cas9
KW - Gene editing
KW - Neurodegeneration
KW - Presenilin
KW - Therapeutics
UR - http://www.scopus.com/inward/record.url?scp=85111514756&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85111514756&partnerID=8YFLogxK
U2 - 10.1016/j.jare.2021.07.001
DO - 10.1016/j.jare.2021.07.001
M3 - Article
C2 - 36100328
AN - SCOPUS:85111514756
VL - 40
SP - 207
EP - 221
JO - Journal of Advanced Research
JF - Journal of Advanced Research
SN - 2090-1232
ER -