Modulation of protein accumulation and uses therefor

Stephanie Rayner (Inventor), Albert Lee (Inventor), Roger Chung (Inventor), Ian Blair (Inventor)

Research output: Patent


Disclosed are compositions and methods for inhibiting abnormal protein accumulation, promoting motor neuron survival, inhibiting motor neuron degeneration and treating neurodegenerative conditions through expression of a nucleic acid sequence encoding cyclin F in motor neurons with an abnormally low level or activity of cyclin F. Also disclosed are methods for identifying agents that promote survival of motor neurons, inhibit degeneration of motor neurons and/or inhibit abnormal protein accumulation in motor neurons, identifying agents that are useful for treating neurodegenerative conditions, diagnosing neurodegenerative conditions, predicting the progression of neurodegenerative conditions, and monitoring the effectiveness of a therapy in reducing the progression of a neurodegenerative condition.
Original languageEnglish
Patent numberEP3535403
IPCC12N 15/861,A61K 48/00,C12Q 1/68
Priority date7/11/16
Publication statusSubmitted - 11 Sept 2019


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