An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model
Marco Peviani, Sabyasachi Das, Janki Patel, Odella C. Jno-Charles, Rajesh Kumar, Ana Zguro, Tyler D. Mathews, Paolo Cabras, Rita Milazzo, Eleonora Cavalca, Valentina Poletti, Alessandra Biffi
This study demonstrates that gene therapy using hematopoietic stem cells can mitigate CLN1 disease symptoms in mice, with enhanced benefits from hPPT1 overexpression and combined transplantation approaches.
EMBO Molecular Medicine · Mar 6, 2023Peer-reviewedNeuroscience