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 Ther
; 27(1-2): 74-84, 2020 02.
Article
in En
| MEDLINE
| ID: mdl-31827259
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Mucopolysaccharidosis I
/
RNA, Untranslated
/
Iduronidase
Limits:
Animals
Language:
En
Journal:
Gene Ther
Journal subject:
GENETICA MEDICA
/
TERAPEUTICA
Year:
2020
Document type:
Article
Affiliation country:
Brasil
Country of publication:
Reino Unido