Monthly Archives: April 2013

Genetic correction of patient-specific LGMD2I iPS cells and the regenerative potential of their skeletal muscle derivatives

Dr. Rita Perlingeiro University of Minnesota (Minneapolis, MN) Project Summary: This grant will support a stem-cell therapy project using genetically-corrected LGMD2I patient cells. In this project, induced pluripotent stem cells (iPS cells) from LGMD2I patients will first be corrected for their genetic defect using novel recombinant DNA techniques. Corrected cells will be then induced […]

Discovery of chemical chaperones for the treatment of FKRP-related limb girdle muscular dystrophy 2I

Dr. Sebahattin Cirak Children’s National Medical Center (Washington, DC) Project Summary: This grant supports drug screening on mutant forms of FKRP, in the search for compounds that restore normal protein function. A library of chemical chaperones will be tested for their ability to stabilize and restore protein folding in FKRP mutated forms. The project […]