1 Grant/ $50,000

Title: Novel ALS Treatments Targeting Muscle Ciliary Neurothrophic Factor Receptor Signaling

Amount: $50,000 – Starr Family Spark Grant in ALS Research

Primary Investigator: John Maclennan, PhD

Category: ALS

Recent discoveries in Dr. MacLennan’s laboratory suggest that: 1) muscle ciliary neurotrophic factor receptor α (CNTFRα) naturally inhibits ALS and 2) increasing muscle CNTFRα could serve as a broadly effective ALS treatment without side effects. These discoveries also suggest that the treatment could start late in the disease process, as is required in humans given ALS’s late diagnosis. Moreover, development of the treatment for clinical use will not depend on the very difficult identification of the potentially many different causes of ALS.

Dr. MacLennan’s laboratory will use the grant funds to optimize this potential treatment by testing several “next generation” adeno-associated virus (AAV) vectors that increase expression of muscle CNTFRα. They will be tested in the most widely used ALS model mice that contain a human ALS-causing mutation and get an ALS-like disease.

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