Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis

Transformative results of adeno-associated virus (AAV) gene therapy in patients with spinal muscular atrophy and Leber’s congenital amaurosis led to approval of the first two AAV products in the United States to treat these diseases. These extraordinary results led to a dramatic increase in the numb...

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Detalles Bibliográficos
Autores principales: Taghian, Toloo, Batista, Ana Rita, Kamper, Sarah, Caldwell, Michael, Lilley, Laura, Li, Hao, Rodriguez, Paola, Mesa, Katerina, Zheng, Shaokuan, King, Robert M., Gounis, Matthew J., Todeasa, Sophia, Maguire, Anne, Martin, Douglas R., Sena-Esteves, Miguel, Meade, Thomas J., Gray-Edwards, Heather L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8517204/
https://www.ncbi.nlm.nih.gov/pubmed/34703836
http://dx.doi.org/10.1016/j.omtm.2021.08.003

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