Associated Papers
AAV-mediated GBA1 and GDNF rescue neurological defects in a murine model of neuronopathic Gaucher disease.
Meng Y et al.
Molecular Therapy. Nucleic Acids, 2025
40206656 (PubMed )
Supplement: mmc2.pdf
Induced Pluripotent Stem Cell Modeling of Gaucher's Disease: What Have We Learned?
Santos DM et al.
International Journal of Molecular Sciences, 2017
Ambroxol chaperone therapy for neuronopathic Gaucher disease: A pilot study.
Narita A et al.
Annals of Clinical and Translational Neurology, 2016
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