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Generation of a monoclonal antibody against FKRP

Dr. Isabelle Richard Généthon (Evry, France)   Project summary: This grant will support the generation of a monoclonal antibody recognizing both the mouse and human forms of FKRP. Once produced, this antibody will be made available to the scientific community.

Generation and characterization of antibodies to alpha-dystroglycan

Dr. Susan Brown Royal Veterinary College (London UK) Dr. Glenn Morris RJAH Orthopaedic Hospital Trust (Oswestry, UK)   Project summary: This grant will support the development of antibodies against different forms of alpha-dystroglycan. Different approaches will be used to generate antibodies that recognize normal and disease forms of alpha-dystroglycan. The grant will support work done … Continue reading “Generation and characterization of antibodies to alpha-dystroglycan”

Development of dystroglycanopathy patient specific cell based assays for drug screening

Dr. Anne Bang Sanford-Burnham Medical Research Institute (La Jolla, CA, USA)   Project summary: This grant will support the development of patient-specific cell lines to be used for drug screening. Skin cells from dystroglycanopathy patients (with different FKRP mutations, or with mutations in POMT1, POMT2, POMGnT or Fukutin genes) will be used to generate several … Continue reading “Development of dystroglycanopathy patient specific cell based assays for drug screening”

Development of Soluble mini-Agrin as a Treatment for Dystroglycanopathies

Dr. Jasbir Seehra Achelois Biosciences (Lexington, MA, USA)   Project summary: This project will test a novel recombinant protein, called Mini-Agrin, as a treatment for dystroglycanopathies. The project is split into 2 phases: the initial phase will test whether mini-agrin can overcome the defect in alpha-dystroglycan caused by FKRP mutations using cell lines. If this … Continue reading “Development of Soluble mini-Agrin as a Treatment for Dystroglycanopathies”

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