Gene replacement therapy for muscular dystrophy
US6262035A · kind A · utility
Assignee
Inventors
Key dates
| Filing date | Oct 1, 1998 |
| Grant date | Jul 17, 2001 |
| Priority date | — |
| Expiry date | Oct 1, 2018 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61K48/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Disclosed is a method for treating a patient suffering from the disease sarcoglycan-deficient limb-girdle muscular dystrophy by gene replacement therapy. Sarcoglycan gene replacement therapy produces extensive long-term expression of the sarcoglycan species which restores the entire sarcoglycan complex, results in the stable association of alph.alpha.-dystroglycan with the sarcolemma, and eliminates the morphological markers of limb-girdle muscular dystrophy. In another aspect, the invention relates to a method for determining a specific defective sarcoglycan species in the tissue of a patient. The method involves culture of muscle cells obtained from the patient, and the independent introduction of expression vectors encoding each of the sarcoglycan species, .alpha., .beta., .gamma., and .delta., into the cultured cells with subsequent assaying for restoration of the dystrophin-glycoprotein complex. In another aspect, the invention relates to a mouse, and cells derived therefrom, homozygous for a disrupted .alpha.-sarcoglycan gene. The disruption prevents the synthesis of functional .alpha.-sarcoglycan in cells of the mouse and results in the mutant mouse having no detectable sarc…
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.