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Emerging Perspectives on Prime Editor Delivery to the Brain.
BenDavid, Eli; Ramezanian, Sina; Lu, Yaoyao; Rousseau, Joël; Schroeder, Avi; Lavertu, Marc; Tremblay, Jacques P.
Affiliation
  • BenDavid E; Laboratory of Biomaterials and Tissue Engineering, Department of Chemical Engineering, Institute of Biomedical Engineering, Polytechnique Montréal, Montréal, QC H3C 3A7, Canada.
  • Ramezanian S; Division of Human Genetics, Centre de Recherche du CHU de Québec-Université Laval, Québec, QC G1V 4G2, Canada.
  • Lu Y; Laboratory of Molecular Genetics and Gene Therapy, Department of Molecular Medicine, Faculty of Medicine, Laval University, Québec, QC G1V 0A6, Canada.
  • Rousseau J; Laboratory of Nanopharmacology and Pharmaceutical Nanoscience, Faculty of Pharmacy, Laval University, Québec, QC G1V 4G2, Canada.
  • Schroeder A; Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa 3525433, Israel.
  • Lavertu M; Division of Human Genetics, Centre de Recherche du CHU de Québec-Université Laval, Québec, QC G1V 4G2, Canada.
  • Tremblay JP; Laboratory of Molecular Genetics and Gene Therapy, Department of Molecular Medicine, Faculty of Medicine, Laval University, Québec, QC G1V 0A6, Canada.
Pharmaceuticals (Basel) ; 17(6)2024 Jun 11.
Article in En | MEDLINE | ID: mdl-38931430
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.
Key words

Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Pharmaceuticals (Basel) Year: 2024 Document type: Article Affiliation country: Canada Country of publication: Switzerland

Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Pharmaceuticals (Basel) Year: 2024 Document type: Article Affiliation country: Canada Country of publication: Switzerland