Invention Grant
- Patent Title: Materials and methods for treating diseases caused by genetic disorders using aminoglycosides and derivatives thereof which exhibit low nephrotoxicity
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Application No.: US14589735Application Date: 2015-01-05
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Publication No.: US09775854B2Publication Date: 2017-10-03
- Inventor: Bruce A. Molitoris , David M. Bedwell , Ruben M. Sandoval
- Applicant: Indiana University Research and Technology Corp.
- Applicant Address: US IN Indianapolis US AL Birmingham
- Assignee: Indiana University Research and Technology Corporation,UAB Research Foundation
- Current Assignee: Indiana University Research and Technology Corporation,UAB Research Foundation
- Current Assignee Address: US IN Indianapolis US AL Birmingham
- Agency: Leydig, Voit & Mayer, Ltd.
- Main IPC: A61K31/7034
- IPC: A61K31/7034 ; A61K31/7036

Abstract:
Various aspects related to the preparation of congeners of the aminoglycosides gentamicin such as the congener C2 and using this compound or derivatives thereof and pharmaceutically active salts to treat diseases that involve genetic mutations which introduce a missense or premature stop codon into a gene. Still other aspects include treating human or animal patients with the gentamicin congener C2 and derivatives and pharmaceutical salt thereof to overcome, or to at least mitigate, the symptoms of disease and disorders such as some forms of Becker's or Duchenne muscular dystrophy, Hurler's Syndrome and Cystic Fibrosis that have as their etiology the presence of a premature stop codon in a gene whose proper expression is necessary for good health.
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