Genome Editing for Mucopolysaccharidoses

Genome Editing for Mucopolysaccharidoses

Genome editing holds the promise of one-off and potentially curative therapies for many patients with genetic diseases. This is especially true for patients affected by mucopolysaccharidoses as the disease pathophysiology is amenable to correction using multiple approaches. Ex vivo and in vivo genom...

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Bibliographic Details
Main Authors: Edina Poletto, Guilherme Baldo, Natalia Gomez-Ospina
Format: Article
Language:English
Published: MDPI AG 2020-01-01
Series:International Journal of Molecular Sciences
Subjects:
genome editing
gene therapy
mucopolysaccharidoses
lysosomal storage disease
crispr/cas9
hurler
hunter
zinc finger nucleases
viral vectors
non-viral vectors
hematopoietic stem cell transplantation
Online Access:https://www.mdpi.com/1422-0067/21/2/500

Internet

https://www.mdpi.com/1422-0067/21/2/500

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