PROJECT

CRISPR-Cas-based gene therapy for PRNP gene silencing in a human prion disease model

  • Start date: 01/10/2024

  • End date: 30/09/2025

  • Project Code: A4348_16386

  • Acronym: Enfermedad priónica humana

PROJECT TYPE

  • Contractes recerca i desenvolupament

Program

Responsible

SHARE

Since the endogenous prion protein plays a central role in the pathogenesis of prion diseases, gene therapy aimed at reducing the expression levels of this protein using CRISPR-Cas technology could represent a promising therapeutic strategy for these diseases and, potentially, for other neurodegenerative diseases such as Alzheimer's and Parkinson's, with similar pathogenic mechanisms.

ENTITATS FINANÇADORES

Convocation

  • Proyectos en enfermedades priónicas 2024 - Enfermedad priónica humana
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