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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5545771/
https://www.ncbi.nlm.nih.gov/pubmed/28808666
http://dx.doi.org/10.1016/j.omtm.2017.07.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5545771/
https://www.ncbi.nlm.nih.gov/pubmed/28808666
http://dx.doi.org/10.1016/j.omtm.2017.07.005

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