CRISPR/Cas9 Editing for Gaucher Disease Modelling

Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by mutations in the acid β-glucosidase gene (GBA1). Besides causing GD, GBA1 mutations constitute the main genetic risk factor for developing Parkinson’s disease. The molecular basis of neurological manifestations in GD...

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Detalles Bibliográficos
Autores principales: Pavan, Eleonora, Ormazabal, Maximiliano, Peruzzo, Paolo, Vaena, Emilio, Rozenfeld, Paula, Dardis, Andrea
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7246564/
https://www.ncbi.nlm.nih.gov/pubmed/32380730
http://dx.doi.org/10.3390/ijms21093268

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7246564/
https://www.ncbi.nlm.nih.gov/pubmed/32380730
http://dx.doi.org/10.3390/ijms21093268

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