Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases

Transthyretin amyloidosis (ATTR) is a progressive and fatal disease caused by transthyretin (TTR) amyloid fibril accumulation in tissues, which disrupts organ function. As the TTR protein is primarily synthesized by the liver, liver transplantation can cure familial ATTR but is not an option for the...

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Detalles Bibliográficos
Autores principales: Greig, Jenny A., Breton, Camilo, Ashley, Scott N., Martins, Kelly M., Gorsuch, Cassandra, Chorazeczewski, Joanna K., Furmanak, Thomas, Smith, Melanie K., Zhu, Yanqing, Bell, Peter, Shoop, Wendy, Li, Hui, Smith, Jeff, Tomberlin, Ginger, Clark, Peter, Mitchell, Thomas W., Buza, Elizabeth L., Yan, Hanying, Jantz, Derek, Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9700363/
https://www.ncbi.nlm.nih.gov/pubmed/36375122
http://dx.doi.org/10.1089/hum.2022.061

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