Author

Vania Broccoli

2 papers indexed here

We haven’t gathered this author’s papers yet. Follow them and we’ll fetch their work.

Not the right person? Other researchers publish under this name.

#gene editing Review Sep 2026

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

#gene editing Open access Sep 2026

Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease benefits a murine model of Alexander disease.

Astrocytes are intriguing therapeutic targets due to their crucial role in initiating and promoting demyelination in the central nervous system. By efficient on-target editing, we can permanently disrupt detrimental genes and restore normal physiological processes. Here, we have optimized an AAV platform to deliver the...

Anne E. J. Hillen, I. Capuano, Barbara Gega et al. · Molecular Therapy · 0 citations