Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies

Lysosomal diseases are a class of genetic disorders predominantly caused by loss of lysosomal hydrolases, leading to lysosomal and cellular dysfunction. Enzyme replacement therapy (ERT), where recombinant enzyme is given intravenously, internalized by cells, and trafficked to the lysosome, has been...

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Detalles Bibliográficos
Autores principales: Baik, Andrew D., Calafati, Philip, Zhang, Xiaoli, Aaron, Nina A., Mehra, Antonia, Moller-Tank, Sven, Miloscio, Lawrence, Praggastis, Maria, Giovannone, Nicholas, Pan, Cheryl, Tang, Yajun, Bridges, Susannah, Mujica, Alejo, Barbounis, Peter, Yanolatos, Jean, Gale, Nicholas, Li, Ning, Kyratsous, Christos A., Schoenherr, Christopher J., Murphy, Andrew J., Economides, Aris N., Cygnar, Katherine D.
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/PMC8636175/
https://www.ncbi.nlm.nih.gov/pubmed/34400331
http://dx.doi.org/10.1016/j.ymthe.2021.08.020

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