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Nature Communications|September 8, 2019
Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type INatalia Gomez-Ospina, Samantha G Scharenberg, Nathalie Mostrel, et al.
Journal of Inherited Metabolic Disease|May 17, 2026
Natural History of Morquio A SyndromeShunji Tomatsu, Kimberly Klipner, Marie Sahou, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|February 18, 2019
Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and FutureMadeleine Taylor, Shaukat Khan, Molly Stapleton, et al.
The American Journal of Tropical Medicine and Hygiene|October 8, 2024
Operational Research to Support Rapid Evidence-Based Responses to Outbreaks: Learnings from COVID-19Anne Hoppe, Pallavi Dani, Grace Mwangoka, et al.
The Journal of Clinical Investigation|June 20, 2025
Neuraminidase 1 secondary deficiency contributes to CNS pathology in neurological mucopolysaccharidoses via brain protein hypersialylationTianMeng Xu, Rachel Heon-Roberts, Travis Moore, et al.
Journal of the International AIDS Society|March 21, 2021
Point-of-care testing can achieve same-day diagnosis for infants and rapid ART initiation: results from government programmes across six African countriesCaroline E Boeke, Jessica Joseph, Melody Wang, et al.
Biorxiv : the Preprint Server for Biology|September 2, 2025
Mono-allelic p.R37H Dehydrodolichyl Diphosphate Synthase variants lead to protein glycosylation defects, aberrant lipid profiles and interneuron scarcity in a novel mouse model of progressive epileptic encephalopathyAfitz Da Silva, Samuel Boris Tene Tadoum, Irena J J Muffels, et al.
Frontiers in Medicine|May 25, 2026
RNA activation as a precision dosing modality: MTL-CEBPA for controlled enzyme elevation in MPS I-HVikash Reebye, Konstantina Skourti-Stathaki, Konstantinos Vanezis, et al.
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