Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal

Infantile malignant osteopetrosis is a devastating disorder of early childhood that is frequently fatal and for which there are only limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative fo...

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Detalles Bibliográficos
Autores principales: Moscatelli, Ilana, Almarza, Elena, Schambach, Axel, Ricks, David, Schulz, Ansgar, Herzog, Christopher D., Henriksen, Kim, Askmyr, Maria, Schwartz, Jonathan D., Richter, Johan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7848732/
https://www.ncbi.nlm.nih.gov/pubmed/33575431
http://dx.doi.org/10.1016/j.omtm.2020.12.009

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