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Article

7T MRI Predicts Amelioration of Neurodegeneration in the Brain after AAV Gene Therapy

24 Dec 2019

Abstract excerpt

GM1 gangliosidosis (GM1) is a fatal neurodegenerative lysosomal storage disease that occurs most commonly in young children, with no effective treatment available. Long-term follow-up of GM1 cats treated by bilateral thalamic and deep cerebellar nuclei (DCN) injection of adeno-associated virus (AAV)-mediated gene therapy has increased lifespan to 8 years of age, compared with an untreated lifespan of ~8 months....

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PMID: 31970203
DOI: 10.1016/j.omtm.2019.11.023

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