| Accession | PRJCA003361 | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| Title | Establishment of humanized muscle-chimeric DMD mice model and the research of CRISPR/Cas9-mediated deficient gene editing and the restoration of long-term expression of dystrophin. | ||||||||
| Relevance | Medical | ||||||||
| Data types |
Exome
Transcriptome or Gene expression Raw sequence reads WES, RNAseq, GUIDE-seq, Nanopore seq, Amplicon-seq |
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| Organisms | Homo sapiens | ||||||||
| Description | Duchenne Muscular Dystrophy (DMD) is a deadly X-linked recessive hereditary disease, which caused by the deficiency of dystrophin arising from mutations of DMD gene. Nowadays, gene therapy is the hotspot for the research of DMD. CRISPR/Cas9 could edit gene defects permanently, and have made progress on mdx mice. But if CRISPR/Cas9 could edit human DMD gene in vivo and restore dystrophin was still unknown. Our previous studies indicated that human-derived MDSCs could differentiate into muscle cells in mice. So we assumed that if we transplant DMD patients' MDSCs to immunodeficient mice, build a humanized muscle-chimeric DMD model mice, and treat the model mice with AAV- CRISPR/Cas9. It is likely to restore dystrophin and function persistently in DMD derived muscle cells. Meanwhile, we could explore the mechanism, efficiency and security of CRISPR/Cas9 in DMD gene editing in vivo. This project could provide important basis for CRISPR/Cas9 in DMD clinical therapy. | ||||||||
| Sample scope | Monoisolate | ||||||||
| Release date | 2020-08-31 | ||||||||
| Grants |
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| Submitter | Xiaoping Li (lixiaoping@mail.sysu.edu.cn) | ||||||||
| Organization | Sun Yat-sen University | ||||||||
| Submission date | 2020-08-31 |