Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector-Reference-Cited by-同舟云学术

Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

Published:2021-03 Issue: Volume:20 Page:312-323
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Dahl Maria^ORCID,Smith Emma M.K.,Warsi Sarah,Rothe Michael,Ferraz Maria J.,Aerts Johannes M.F.G.,Golipour Azadeh,Harper Claudia,Pfeifer Richard,Pizzurro Daniella,Schambach Axel,Mason Chris,Karlsson Stefan

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference36 articles.

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3. A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments;Stirnemann;Int. J. Mol. Sci.,2017

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5. The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease;Hollak;Best Pract. Res. Clin. Endocrinol. Metab.,2015

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