Zobrazeno 1 - 1
of 1
pro vyhledávání: '"Emma M.K. Smith"'
Autor:
Maria Dahl, Emma M.K. Smith, Sarah Warsi, Michael Rothe, Maria J. Ferraz, Johannes M.F.G. Aerts, Azadeh Golipour, Claudia Harper, Richard Pfeifer, Daniella Pizzurro, Axel Schambach, Chris Mason, Stefan Karlsson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 20, Iss , Pp 312-323 (2021)
Gaucher disease type 1 (GD1) is an inherited lysosomal disorder with multisystemic effects in patients. Hallmark symptoms include hepatosplenomegaly, cytopenias, and bone disease with varying degrees of severity. Mutations in a single gene, glucosida
Externí odkaz:
https://doaj.org/article/07372d5f46f04d9c947be435687180aa