Niemann-Pick disease type C1

3 papers

Animals3

Latest

Human Molecular Genetics1 Sep

AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease

Experiments in miceInterventions

Gene6 Dec

In Niemann-Pick C1 mouse models, glial-only expression of the normal gene extends survival much further than do changes in genetic background or treatment with hydroxypropyl-beta-cyclodextrin

Experiments in miceInterventions

Human Molecular Genetics1 Jan

Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

Experiments in miceInterventions