Author
Children's NationalORCID 0000-0002-1999-336XUnclaimed ORCID profile · follow to get notified of new preprints
Mutual co-authors in cluster
4 papers
Recent·Most cited
Connor J. Lewis, Precilla D’Souza, Jean M. Johnston…·Cynthia J. Tifft
This phase 1/2 trial evaluates AAV9 gene therapy for GM1 gangliosidosis Type II, showing safety and some biochemical and neuroimaging improvements.
Clara M. Abdelmalek, Shriya Singh, Blain Fasil…·Youssef A. Kousa
The PING Consortium builds a genomic data repository to study genetic modifiers of prenatal viral infections' effects on brain development and neurodevelopmental outcomes.
Precilla D’Souza, Cristan Farmer, Jean M. Johnston…·Cynthia J. Tifft
This study presents a 10-year prospective analysis of type II GM1 gangliosidosis, detailing disease progression and phenotypic features in 41 patients.
Ben Shofty, Eyal Bergmann, Gil Zur…·Itamar Kahn
This study shows that Nf1 loss-of-function disrupts corticocortical and corticostriatal functional connectivity in both human NF1 patients and Nf1+/- mice.