What if we could treat ALS not by replacing damaged neurons, but by correcting the molecular errors that prevent them from functioning properly? That question inspired SplicePatch, our proposed RNA-based therapy for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease that gradually destroys the motor neurons responsible for movement, speech, and breathing. We were particularly interested in a common feature of ALS: the dysfunction of a protein called TDP-43. Normally, TDP-43 helps regulate how RNA is processed. When it becomes depleted from the nucleus, important genes such as STMN2 and UNC13A can be incorrectly spliced, reducing proteins essential for neuronal repair and communication. We wanted to explore whether correcting both RNA-processing errors could help preserve motor-neuron function, while also making treatment delivery more manageable for patients.
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