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Mucolipidosis Collaborative Research Network (MCRN)

14 Sep, 2020

Accelerating a Treatment for GNTAB-Related Disorders 
Richard Steet, Ph.D.
Jennifer Klein, MS

One year since the formation of the MCRN, the network continues to meet monthly, with the primary function to coordinate several labs focused on ML research. The Network executes systematic and standardized testing of different treatment ideas in common animal models for ML II, ML II/III and ML III alpha/beta. The Network has 12 members including researchers, clinicians, National MPS Society, ISMRD, and one patient scientist.

The MCRN treatment plan and workflow includes the following areas: expanding feline ML colony at UPENN and UC-Davis; evaluating AAV9-GNTAB treated cats in different labs; focusing on skeletal and cardiac systems but CNS also will be studied; treating ML cats with small molecule therapies and evaluate efficacy broadly in all affected tissues; and establishing a workflow for the testing of any new therapies with positive cell-based or zebra-based findings. Treatment modalities for ML include gene therapy; cathepsin inhibition; modulation of TGFƁ signaling; and induction of lysosomal clearance.

The MCRN construct will accelerate research and provide accountability to meet set goals. Demonstrating their effectiveness, the Network will apply for larger grants through the NIH. Finally, conditions like ML require all possible therapeutic ideas to be rigorously tested in proven animal models.