Undergraduate Summer Student Job Details

Molecular Mechanisms of Neuromuscular Diseases and Antisense/Gene-editing Therapeutics
Toshifumi Yokota, PhD
8-31 Medical Sciences Building
780 492.1102
1) Antisense and Genome Editing Therapies
Antisense-mediated therapy is an exciting new approach to treating diseases using DNA-like molecules. These molecules act like a stitch or Band-Aid to mitigate the effects of genetic mutations and restore the gene function. By utilizing integrative experimental and computational approaches, such as antisense oligonucleotides, CRISPR/Cas9 genome editing, and machine-learning, the focuses of our group are to develop novel personalized molecular therapies for neuromuscular and musculoskeletal diseases.
2) Role of Water Channel Aquaporins in Muscle and Brain
A water channel Aquaporin-4 (AQP4) is known to selectively express in the fast-twitch skeletal muscle fibres and at the perivascular blood-brain-barrier (BBB) in the brain; however its physiological function remains poorly understood. The goal of our research program is to characterize the role of AQP4 in response to the muscle exercise and fatigue in muscles and brains.
Medical Genetics

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