Daniela Ungaro

TL;DR: It is demonstrated here that HSC gene therapy, based on lentiviral vectors, completely corrects disease manifestations in the mouse model and provides evidence of an efficacious treatment for MPS I Hurler patients, warranting future development toward clinical testing.

TL;DR: Findings indicate that ASC may promote neuroprotection either directly and/or by modulating the secretome of local glial cells toward a neuroprotective phenotype and encourages the use of ASC in human pathologies, in which current therapies are not able to maintain a satisfying neurological functional status.

TL;DR: Reduction of the CHOP target Gadd34 restores motor function in P0S63del mice with demyelinating neuropathy and reduces the need for CHOP therapy in these mice.

TL;DR: Jab1 constitutes a regulatory molecule that integrates laminin211 signals in Schwann cells to govern cell cycle, cell number, and differentiation.

TL;DR: It is shown that Perk loss of function in Schwann cells restores myelination without diminishing accumulation of P0 or markers of ER stress, suggesting that PerK may modulate myelinations through a pathway independent of the UPR.