CRISPR/Cas9 gene editing: New hope for Alzheimer's disease therapeutics

Shanu Bhardwaj, Kavindra Kumar Kesari, Mahesh Rachamalla, Shalini Mani, Ghulam Md Ashraf, Saurabh Kumar Jha, Pravir Kumar, Rashmi K. Ambasta, Harish Dureja, Hari Prasad Devkota, Gaurav Gupta, Dinesh Kumar Chellappan, Sachin Kumar Singh, Kamal Dua, Janne Ruokolainen, Mohammad Amjad Kamal, Shreesh Ojha, Niraj Kumar Jha

Research output: Contribution to journalArticlepeer-review

38 Citations (Scopus)


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.

Original languageEnglish
Pages (from-to)207-221
Number of pages15
JournalJournal of Advanced Research
Publication statusPublished - Sept 2022


  • APP
  • Alzheimer's disease
  • CRISPR/Cas9
  • Gene editing
  • Neurodegeneration
  • Presenilin
  • Therapeutics

ASJC Scopus subject areas

  • General


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