Emerging Perspectives on Prime Editor Delivery to the Brain-Reference-Cited by-同舟云学术

Emerging Perspectives on Prime Editor Delivery to the Brain

Published:2024-06-11 Issue:6 Volume:17 Page:763
ISSN:1424-8247
Container-title:Pharmaceuticals
language:en
Short-container-title:Pharmaceuticals

Author:

BenDavid Eli¹²³⁴⁵^ORCID,Ramezanian Sina²³,Lu Yaoyao²³^ORCID,Rousseau Joël²,Schroeder Avi⁶,Lavertu Marc¹^ORCID,Tremblay Jacques P.²³^ORCID

Affiliation:

1. Laboratory of Biomaterials and Tissue Engineering, Department of Chemical Engineering, Institute of Biomedical Engineering, Polytechnique Montréal, Montréal, QC H3C 3A7, Canada

2. Division of Human Genetics, Centre de Recherche du CHU de Québec—Université Laval, Québec, QC G1V 4G2, Canada

3. Laboratory of Molecular Genetics and Gene Therapy, Department of Molecular Medicine, Faculty of Medicine, Laval University, Québec, QC G1V 0A6, Canada

4. Laboratory of Nanopharmacology and Pharmaceutical Nanoscience, Faculty of Pharmacy, Laval University, Québec, QC G1V 4G2, Canada

5. Rappaport Faculty of Medicine, Technion—Israel Institute of Technology, Haifa 3525433, Israel

6. Laboratory for Targeted Drug Delivery and Personalized Medicine Technologies, Department of Chemical Engineering, Technion—Israel Institute of Technology, Haifa 3200003, Israel

Abstract

Prime editing shows potential as a precision genome editing technology, as well as the potential to advance the development of next-generation nanomedicine for addressing neurological disorders. However, turning in prime editors (PEs), which are macromolecular complexes composed of CRISPR/Cas9 nickase fused with a reverse transcriptase and a prime editing guide RNA (pegRNA), to the brain remains a considerable challenge due to physiological obstacles, including the blood–brain barrier (BBB). This review article offers an up-to-date overview and perspective on the latest technologies and strategies for the precision delivery of PEs to the brain and passage through blood barriers. Furthermore, it delves into the scientific significance and possible therapeutic applications of prime editing in conditions related to neurological diseases. It is targeted at clinicians and clinical researchers working on advancing precision nanomedicine for neuropathologies.

Funder

Canadian Institute of Heath Research

Publisher

MDPI AG

Link

https://www.mdpi.com/1424-8247/17/6/763/pdf

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