Duchenne Muscular Dystrophy (DMD) Models
Duchenne muscular dystrophy (DMD) is a severe X-linked recessive disorder characterized by progressive muscle weakness and degenerative neurological symptoms, predominantly affecting males due to a mutation in the DMD gene on the X chromosome. Dystrophin protein is located at the muscle sarcolemma and plays a critical role in maintaining the stability of the muscle sarcolemma. The mutation rate of the DMD gene is relatively high, with exon deletion being the most common mutation, accounting for 55% to 65% of cases according to some disease research. Gene therapy and exon-skipping approaches are emerging therapeutic research directions for DMD.
Novel treatments for DMD cannot be evaluated without the aid of appropriate animal models. In pursuit of understanding the pathogenesis and therapeutic targets of DMD, GemPharmatech has developed a variety of DMD models utilizing gene editing technology independently. These models can be applied to drug screening and disease mechanism research.
|
Strain No.
|
Strain Name | Strain Type | Description |
|---|---|---|---|
| T049591 | B6-Dmd Del52 | Knock Out | The B6-Dmd Del52 mouse model of was constructed by deleting exon 52 of the mouse Dmd gene. |
| T056004 | B6-Dmd Del51 | Knock Out | The B6-Dmd Del51 mouse model of was constructed by deleting exon 51 of the mouse Dmd gene. |
| T014593 | Dmd-C3197T | Point Mutation | Male and female mice are on B10 background and exhibit late-onset DMD with significant motor impairment and fiber degeneration at 8 months of age. |
| T056002 | B6-Dmd Del45 | Knock Out | The B6-Dmd Del45 mouse model of was constructed by deleting exon 45 of the mouse Dmd gene. |
| T056003 | B6-Dmd Del50 | Knock Out | The B6-Dmd Del50 mouse model of was constructed by deleting exon 50 of the mouse Dmd gene. |
| T003035 | B10-Dmd-KO | Knock Out | Male mice are on B10 background and exhibit early-onset DMD with muscular lesions (muscle fiber atrophy, inflammatory cell infiltration, etc.) at 9 weeks. |