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Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis

Efficient disease-modifying treatments for Alzheimer disease, the most common form of dementia, have yet to be established. Gene therapy has the potential to provide the long-term production of therapeutic in the brain following a single administration. However, the blood-brain barrier poses a chall...

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Detalles Bibliográficos
Autores principales: Kofoed, Rikke Hahn, Heinen, Stefan, Silburt, Joseph, Dubey, Sonam, Dibia, Chinaza Lilian, Maes, Miriam, Simpson, Elizabeth M., Hynynen, Kullervo, Aubert, Isabelle
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8560718/
https://www.ncbi.nlm.nih.gov/pubmed/34761053
http://dx.doi.org/10.1016/j.omtm.2021.10.001