A scalable, GMP-compatible, autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa

BACKGROUND. Gene editing in induced pluripotent stem (iPS) cells has been hailed to enable new cell therapies for various monogenetic diseases including dystrophic epidermolysis bullosa (DEB). However, manufacturing, efficacy and safety roadblocks have limited the development of genetically correcte...

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Detalles Bibliográficos
Autores principales: Neumayer, Gernot, Torkelson, Jessica L., Li, Shengdi, McCarthy, Kelly, Zhen, Hanson H., Vangipuram, Madhuri, Jackow, Joanna, Rami, Avina, Hansen, Corey, Guo, Zongyou, Gaddam, Sadhana, Pappalardo, Alberto, Li, Lingjie, Cramer, Amber, Roy, Kevin R., Nguyen, Thuylinh Michelle, Tanabe, Koji, McGrath, Patrick S., Bruckner, Anna, Bilousova, Ganna, Roop, Dennis, Bailey, Irene, Tang, Jean Y., Christiano, Angela, Steinmetz, Lars M., Wernig, Marius, Oro, Anthony E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cold Spring Harbor Laboratory 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10002612/
https://www.ncbi.nlm.nih.gov/pubmed/36909618
http://dx.doi.org/10.1101/2023.02.28.529447

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