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.
| Original language | English |
|---|---|
| Article number | 763 |
| Journal | Pharmaceuticals |
| Volume | 17 |
| Issue number | 6 |
| DOIs | |
| State | Published - 1 Jun 2024 |
| Externally published | Yes |
Keywords
- CRISPR/Cas9
- drug delivery system
- gene therapy
- genetic engineering
- nanomaterial
- neurology
- personalized nanomedicine
- prime editing
- targeted drug delivery
ASJC Scopus subject areas
- Molecular Medicine
- Pharmaceutical Science
- Drug Discovery