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My group’s research focuses on improving and designing new therapies for motor neuron disease spinal muscular atrophy (SMA) and muscular dystrophies Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1). These disorders are treatable with antisense oligonucleotides (ASOs) to correct the dysfunctional mRNA. The team focuses on the targeted delivery of these therapies using a peptide and antibody delivery vehicles towards skeletal muscle and central nervous system tissues. We work with academic and industrial collaborators to advance this work to early clinical development.
Cell penetrating peptides (CPPs) are short cationic or amphipathic peptides that actively and passively penetrate the cell membrane for effective intracellular drug delivery. These CPP-ASO conjugates transport ASOs into skeletal muscle, heart, brain and spinal cord; tissues which are otherwise impenetrable without invasive local administrations. The most recent designs are focused on (1) increasing safety and tolerability and (2) directing a more tissue specific uptake to reduce off target effects.
Cell penetrating peptides (CPPs) are short cationic or amphipathic peptides that actively and passively penetrate the cell membrane for effective intracellular drug delivery. These CPP-ASO conjugates transport ASOs into skeletal muscle, heart, brain and spinal cord; tissues which are otherwise impenetrable without invasive local administrations. The most recent designs are focused on (1) increasing safety and tolerability and (2) directing a more tissue specific uptake to reduce off target effects.
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EMBO molecular medicineno. 11 (2023): n/a-n/a
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EMBO Molecular Medicineno. 11 (2023)
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bioRxiv (Cold Spring Harbor Laboratory) (2022)
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