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Central nervous system (CNS) neurons fail to regenerate after CNS injuries and in neurodegenerative diseases, leading to disabling functional deficits. Regenerating CNS neurons could restore these lost brain connections and functions.

Breakthrough Regeneration Discovery

Dr. Zhigang He (Boston Children’s Hospital and Harvard) performed a genetic screen of tumor suppressors by in vivo knockout for neuron regeneration after optic nerve crush (ONC) injury, and discovered that deletion of phosphatase and tensin homolog (PTEN) enabled unprecedented regeneration of CNS neurons (published in Science and Nature). Since then, Dr. He, Dr. Oswald Steward (University of California, Irvine) and many others have used PTEN-inhibition to induce neuron regeneration in other models of CNS injury (e.g. spinal cord injury).

Unlocking Intrinsic Regenerative Ability of Neurons

Dr. He’s discovery was the first evidence that manipulating an endogenous gene within CNS neurons can unlock an intrisic regenerative ability, leading to re-growth of damaged neurons. In addition to PTEN, a master negative regulator of cell growth, AXONIS Therapeutics has a number of new, proprietary neuroregeneration gene targets, which were identified through a recent, unpublished genome-wide AAV-CRISPR screen in the mouse ONC model.

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