Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts

Autosomal recessive osteopetrosis is a human bone disease mainly caused by TCIRG1 gene mutations that prevent osteoclasts resorbing activity, recapitulated by the oc/oc mouse model. Bone marrow transplantation is the only available treatment, limited by the need for a matched donor. The use of induc...

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Publicado no:Stem Cell Reports
Main Authors: Neri, Tui, Muggeo, Sharon, Paulis, Marianna, Caldana, Maria Elena, Crisafulli, Laura, Strina, Dario, Focarelli, Maria Luisa, Faggioli, Francesca, Recordati, Camilla, Scaramuzza, Samantha, Scanziani, Eugenio, Mantero, Stefano, Buracchi, Chiara, Sobacchi, Cristina, Lombardo, Angelo, Naldini, Luigi, Vezzoni, Paolo, Villa, Anna, Ficara, Francesca
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4624934/
https://ncbi.nlm.nih.gov/pubmed/26344905
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2015.08.005
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