CRISPR-Cas9 in Alzheimer's disease: Therapeutic trends, modalities, and challenges

Leena Chacko; Anupama Chaudhary; Birbal Singh; Saikat Dewanjee; Ramesh Kandimalla

doi:10.1016/j.drudis.2023.103652

CRISPR-Cas9 in Alzheimer's disease: Therapeutic trends, modalities, and challenges

Drug Discov Today. 2023 Aug;28(8):103652. doi: 10.1016/j.drudis.2023.103652. Epub 2023 Jun 7.

Authors

Leena Chacko¹, Anupama Chaudhary², Birbal Singh³, Saikat Dewanjee⁴, Ramesh Kandimalla⁵

Affiliations

¹ BioAnalytical Lab, Meso Scale Discovery, 1601 Research Blvd, Rockville, MD, USA.
² Orinin-BioSystems, LE-52, Lotus Road 4, CHD City, Karnal, Haryana 132 001, India.
³ ICAR-Indian Veterinary Research Institute (IVRI), Regional Station, Palampur, Himachal Pradesh 176 061, India.
⁴ Advanced Pharmacognosy Research Laboratory, Department of Pharmaceutical Technology, Jadavpur University, Kolkata 700 032, India. Electronic address: saikat.dewanjee@jadavpuruniversity.in.
⁵ Department of Biochemistry, Kakatiya Medical College, Warangal 506 007, Telangana, India; Department of Applied Biology, CSIR-Indian Institute of Technology, Uppal Road, Tarnaka, Hyderabad 500 007, India. Electronic address: ramesh.kandimalla@gmail.com.

PMID: 37290639
DOI: 10.1016/j.drudis.2023.103652

Abstract

Alzheimer's disease (AD) is a progressive neurodegenerative disorder with no known cure, which has prompted the exploration of novel therapeutic approaches. The clustered regularly interspaced palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) tool has generated significant interest for its potential in AD therapeutics by correcting faulty genes. Our report comprehensively reviews emerging applications for CRISPR-Cas9 in developing in vitro and in vivo models for AD research and therapeutics. We further assess its ability to identify and validate genetic markers and potential therapeutic targets for AD. Moreover, we review the current challenges and delivery strategies for the in vivo application of CRISPR-Cas9 in AD therapeutics.

Keywords: Alzheimer’s disease; CRISPR-Cas9; Gene editing; Therapeutics.

Publication types

Review

MeSH terms

Alzheimer Disease* / drug therapy
Alzheimer Disease* / genetics
CRISPR-Cas Systems* / genetics
Gene Editing
Genetic Therapy
Humans