CRISPR-mediated megabase-scale transgene de-duplication to generate a functional single-copy full-length humanized DMD mouse model
Our unique hDMDTgSc mouse model simulates the true copy number of the DMD gene, and can potentially be used for the further generation of DMD disease models that would be better suited for the pre-clinical assessment and development of sequence specific CRISPR therapies.
Authors
- Yu Chinn Joshua Chey (ORCID: https://orcid.org/0000-0001-7553-5163)
- Sandra Piltz (ORCID: https://orcid.org/0000-0003-3386-8140)
- Jayshen Arudkumar (ORCID: https://orcid.org/0000-0002-4856-6332)
- Fatwa Adikusuma (ORCID: https://orcid.org/0000-0003-2163-0514)
- Mark Corbett (ORCID: https://orcid.org/0000-0001-9298-3072)
- Paul Q. Thomas (ORCID: https://orcid.org/0000-0002-5002-5770)
Institutions
- South Australian Health and Medical Research Institute (AU)
- AGRF Ltd (AU)
- The University of Adelaide (AU)
Publication Details
- Journal
- BMC Biology
- Published
- 2024-09-27
- DOI
- https://doi.org/10.1186/s12915-024-02008-7
- Citations
- 4
- Primary Topic
- CRISPR and Genetic Engineering
- Type
- article
- Field-Weighted Citation Impact
- 0.94
Funders
- South Australian Health and Medical Research Institute
- Australian Government
- University of Adelaide