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 Emanuel, Peruzzo, Paolo, Vaena, Emilio, Rozenfeld, Paula Adriana, Dardis, Andrea
Formato: Articulo
Lenguaje:Inglés
Publicado: 2020
Materias:
Biología
Gaucher disease
Cellular model
Acid β-glucosidase
Crispr/cas9
Unfolded protein response
Neuroinflammation
Α-synuclein
High-throughput drug screenings
Acceso en línea:http://sedici.unlp.edu.ar/handle/10915/107275
http://europepmc.org/backend/ptpmcrender.fcgi?accid=PMC7246564&blobtype=pdf
Aporte de:
SEDICI (UNLP) de Universidad Nacional de La Plata

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