Gene Therapy for ALS
June 30, 2020 | Terry Sharrer
While it has only been demonstrated in mice, researchers at the University of California-San Diego Medical School report: “A one-time injection of a gene-silencing RNA delivered by an adeno-associated virus (AAV) vector into the spinal cord prevented the onset of ALS in presymptomatic mice, and it blocked disease progression in rodents that had already developed symptoms.” And, “Further analysis showed that the therapy suppressed the accumulation of misfolded SOD1 protein and almost completely preserved motor neuron cells.” MORE
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