Elizabeth M. Eultgen

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

S. Ekins, C. Whitelaw, S. Lillico, 2022, The Journal of clinical investigation.

Cortical interneuron loss and seizure generation as novel clinically relevant disease phenotypes in Cln2R207X mice

J. Cooper, M. Sands, N. Rensing, 2022, bioRxiv.

Enforced differentiation of Dnmt3a-null bone marrow leads to failure with c-Kit mutations driving leukemic transformation.

Christopher A. Miller, J. Klco, J. Hundal, 2014, Blood.

Dnmt3a Regulates T-cell Development and Suppresses T-ALL Transformation

T. Druley, M. Figueroa, G. Challen, 2017, Leukemia.

Title: Bowel dysmotility and enteric neuron degeneration in lysosomal storage disease mice is prevented by gene therapy Short title: Treatment of enteric disease by gene therapy

J. Cooper, R. Heuckeroth, M. Sardiello, 2023 .

Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model

O. Danos, J. Cooper, M. Sands, 2023, The Journal of clinical investigation.