Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Gene Therapy ◽  
2019 ◽  
Vol 27 (1-2) ◽  
pp. 74-84 ◽  
Author(s):  
Roselena Silvestri Schuh ◽  
Esteban Alberto Gonzalez ◽  
Angela Maria Vicente Tavares ◽  
Bruna Gazzi Seolin ◽  
Lais de Souza Elias ◽  
...  
Keyword(s):  
Bone Disease ◽  
Gene Editing ◽  
Mucopolysaccharidosis I

scholarly journals Postnatal progression of bone disease in the cervical spines of mucopolysaccharidosis I dogs

Bone ◽  
2013 ◽  
Vol 55 (1) ◽  
pp. 78-83 ◽  
Author(s):  
Joseph A. Chiaro ◽  
Matthew D. Baron ◽  
Chelsea M. del Alcazar ◽  
Patricia O'Donnell ◽  
Eileen M. Shore ◽  
...  
Keyword(s):  
Bone Disease ◽  
Mucopolysaccharidosis I

Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system

2022 ◽  
Author(s):  
Luisa Natalia Pimentel Vera ◽  
Roselena Silvestri Schuh ◽  
Flavia Nathielly Silveira Fachel ◽  
Edina Poletto ◽  
Eduarda Piovesan ◽  
...  
Keyword(s):  
Gene Editing ◽  
Nasal Delivery ◽  
Mucopolysaccharidosis I
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