Transcutaneous ultrasound-mediated gene delivery into canine livers achieves therapeutic levels of factor VIII expression

A safe, effective, and inclusive gene therapy will significantly benefit a large population of patients with hemophilia. We used a minimally invasive transcutaneous ultrasound-mediated gene delivery (UMGD) strategy combined with microbubbles (MBs) to enhance gene transfer into 4 canine livers. A mix...

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Detalles Bibliográficos
Autores principales: Manson, Megan A., Zhang, Feng, Novokhodko, Alexander, Chen, Chun-Yu, Parker, Maura, Loeb, Keith R., Kajimoto, Masaki, Campbell, Carley, Storb, Rainer F., Miao, Carol H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Hematology 2022
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9631573/
https://www.ncbi.nlm.nih.gov/pubmed/35427415
http://dx.doi.org/10.1182/bloodadvances.2021006016

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9631573/
https://www.ncbi.nlm.nih.gov/pubmed/35427415
http://dx.doi.org/10.1182/bloodadvances.2021006016

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