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Emerging perspectives on prime editor delivery to the brain

Eli BenDavid, Sina Ramezanian, Yaoyao Lu, Joël Rousseau, Avi Schroeder, Marc Lavertu and Jacques P. Tremblay

Article (2024)

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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.

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Additional Information: Groupe de recherche: Laboratory of Biomaterials and Tissue Engineering
Subjects: 1800 Chemical engineering > 1800 Chemical engineering
1900 Biomedical engineering > 1900 Biomedical engineering
1900 Biomedical engineering > 1901 Biomedical technology
Department: Department of Mechanical Engineering
Institut de génie biomédical
Research Center: Other
Funders: Canadian Institute of Heath Research
Grant number: 185747
PolyPublie URL: https://publications.polymtl.ca/58613/
Journal Title: Pharmaceuticals (vol. 17, no. 6)
Publisher: MDPI
DOI: 10.3390/ph17060763
Official URL: https://doi.org/10.3390/ph17060763
Date Deposited: 26 Jun 2024 12:51
Last Modified: 26 Sep 2024 12:51
Cite in APA 7: BenDavid, E., Ramezanian, S., Lu, Y., Rousseau, J., Schroeder, A., Lavertu, M., & Tremblay, J. P. (2024). Emerging perspectives on prime editor delivery to the brain. Pharmaceuticals, 17(6), 763 (42 pages). https://doi.org/10.3390/ph17060763

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