Targeted Epigenetic Reactivation strategies as new treatments for Prader-Willi Syndrome: Achievements and Challenges.
Key translational challenges are discussed, focusing on the imperative to optimize delivery vectors for widespread central nervous system distribution, the necessity of advanced human-derived neuronal models to validate functional recovery, and the critical need to prevent iatrogenic Angelman Syndrome caused by UBE3A i...
S. Alberti, Irina Cutei, Giacomo Costa Broccardi et al.
· Molecular Therapy · 0 citations