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Emerging Perspectives on Prime Editor Delivery to the Brain.

Eli BenDavid1,2,3,4,5, Sina Ramezanian2,3, Yaoyao Lu2,3

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

Pharmaceuticals (Basel, Switzerland)
|June 27, 2024
PubMed
Summary
This summary is machine-generated.

Prime editing offers precision genome editing for neurological disorders. This review explores strategies for delivering prime editors (PEs) across the blood-brain barrier (BBB) for advanced nanomedicine therapies.

Keywords:
CRISPR/Cas9drug delivery systemgene therapygenetic engineeringnanomaterialneurologypersonalized nanomedicineprime editingtargeted drug delivery

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Area of Science:

  • Biomedical Engineering
  • Neuroscience
  • Genetics

Background:

  • Prime editing (PE) is a powerful genome editing technology with potential for neurological disease treatment.
  • Delivering prime editors (PEs) to the brain is challenging due to the blood-brain barrier (BBB).

Purpose of the Study:

  • To provide an overview of current technologies and strategies for brain delivery of prime editors.
  • To discuss the therapeutic potential of prime editing for neurological disorders.

Main Methods:

  • Review of recent literature on prime editing delivery systems.
  • Analysis of strategies for overcoming physiological barriers like the BBB.
  • Exploration of nanomedicine approaches for brain targeting.

Main Results:

  • Emerging technologies show promise for enhanced PE delivery across the BBB.
  • Various strategies are being developed to improve PE brain penetration and targeting.
  • Prime editing holds significant therapeutic potential for diverse neuropathologies.

Conclusions:

  • Effective delivery of prime editors to the brain is crucial for advancing nanomedicine in treating neurological diseases.
  • Continued research into delivery systems and therapeutic applications is essential for clinical translation.