Skip to main content

Dr. Rüdiger Horstkorte & Dr. Kaya Bork

Institute for Physiological Chemistry, Martin-Luther-Universität Halle–Wittenberg

Development of a sensitive and robust enzyme assays for GNE (UDP-N-acetylmannosamine

2-epimerase/N-acetylmannosamine kinase)

 

Dr. Kelly Crowe

Department of BiologySchool of Behavioral & Natural Sciences Mount St. Joseph University

Evaluation of Lectin Staining Biomarkers in Pre-Clinical Models of GNEM

 

Dr. Julie Crudele

Department of Neurology, University of Washington

Development and preclinical assessment of AAV-GNE gene therapy

 

Dr.  Monkol Lek

Department of Genetics, Yale School of Medicine

High throughput genetic screening assay for GNE

 

Dr. Paul Martin

Center for Gene Therapy, Abigail Wexner Research Institute at Nationwide Children's Hospital

Development and gene therapy testing of a new disease model for GNE myopathy

 

Dr. Stella Mitrani-Rosenbaum

Goldyne Savad Institute of Gene Therapy, Hadassah Hebrew University Medical Center

GNE Myopathy: Establishment of Gne KO muscle cells for the assessment of GNE related biomarkers

 

Dr. Stella Mitrani-Rosenbaum

Goldyne Savad Institute of Gene Therapy, Hadassah Hebrew University Medical Center

GNE Myopathy: GneKO muscle conditional mouse for the assessment of AAVGne gene therapy

 

Dr. Wakako Yoshioka

Department of Neuromuscular Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry

Activation of endogenous mutated GNE product by small compound