In silico analysis of potential off-target sites to gene editing for Mucopolysaccharidosis type I us...
In silico analysis of potential off-target sites to gene editing for Mucopolysaccharidosis type I using the CRISPR/Cas9 system: Implications for population-specific treatments
About this item
Full title
Author / Creator
Publisher
United States: Public Library of Science
Journal title
Language
English
Formats
Publication information
Publisher
United States: Public Library of Science
Subjects
More information
Scope and Contents
Contents
Mucopolysaccharidosis type I (MPS I) is caused by alpha-L-iduronidase deficiency encoded by the
IDUA
gene. Therapy with CRISPR/Cas9 is being developed for treatment, however a detailed investigation of off-target effects must be performed. This study aims to evaluate possible off-targets for a sgRNA aiming to correct the most common variant f...
Alternative Titles
Full title
In silico analysis of potential off-target sites to gene editing for Mucopolysaccharidosis type I using the CRISPR/Cas9 system: Implications for population-specific treatments
Authors, Artists and Contributors
Author / Creator
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_plos_journals_2622603149
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2622603149
Other Identifiers
ISSN
1932-6203
E-ISSN
1932-6203
DOI
10.1371/journal.pone.0262299
