Effective Small Interfering RNA Therapy to Treat CLCN7-dependent Autosomal Dominant Osteopetrosis Type 2

In about 70% of patients affected by autosomal dominant osteopetrosis type 2 (ADO2), osteoclast activity is reduced by heterozygous mutations of the CLCN7 gene, encoding the ClC-7 chloride/hydrogen antiporter. CLCN7(G215R)-, CLCN7(R767W)-, and CLCN7(R286W)-specific siRNAs silenced transfected mutant...

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Detalles Bibliográficos
Autores principales: Capulli, Mattia, Maurizi, Antonio, Ventura, Luca, Rucci, Nadia, Teti, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4877447/
https://www.ncbi.nlm.nih.gov/pubmed/26325626
http://dx.doi.org/10.1038/mtna.2015.21

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4877447/
https://www.ncbi.nlm.nih.gov/pubmed/26325626
http://dx.doi.org/10.1038/mtna.2015.21

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