Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy

GM1 gangliosidosis is a fatal lysosomal disorder, for which there is no effective treatment. Adeno-associated virus (AAV) gene therapy in GM1 cats has resulted in a greater than 6-fold increase in lifespan, with many cats remaining alive at >5.7 years of age, with minimal clinical signs. Glycolip...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Mol Ther Methods Clin Dev
Prif Awduron: Gray-Edwards, Heather L., Jiang, Xuntian, Randle, Ashley N., Taylor, Amanda R., Voss, Taylor L., Johnson, Aime K., McCurdy, Victoria J., Sena-Esteves, Miguel, Ory, Daniel S., Martin, Douglas R.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society of Gene & Cell Therapy 2017
Pynciau:
Original Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC5545771/
https://ncbi.nlm.nih.gov/pubmed/28808666
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.07.005
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