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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Detalhes bibliográficos

Principais autores:	Rikke Hahn Kofoed, Stefan Heinen, Joseph Silburt, Sonam Dubey, Chinaza Lilian Dibia, Miriam Maes, Elizabeth M. Simpson, Kullervo Hynynen, Isabelle Aubert
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Elsevier 2021-12-01
coleção:	Molecular Therapy: Methods & Clinical Development
Assuntos:	focused ultrasound microbubbles blood-brain barrier adeno-associated virus rAAV-PHP.B TgCRND8
Acesso em linha:	http://www.sciencedirect.com/science/article/pii/S2329050121001583
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