Author

Hélène Puccio

h-index 4 View on Semantic Scholar

We have 1 of 11 papers

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.

Aug 2026

Physiologically Regulated Frataxin Gene Replacement Restores Neurological Function in a Mouse Model of Friedreich Ataxia.

It is demonstrated that physiologically regulated FXN replacement is sufficient to achieve substantial functional rescue in FA, supporting a gene therapy strategy based on a transgene expression driven by endogenous regulatory elements.

Federica Pilotto, Laure Dall'Agnol, L. Reutenauer et al. · Human Gene Therapy · 0 citations