📰 From the Field: Gene supplementation for LBSL
Annals of Neurology shows AAV9-DARS2 improves mitochondrial function and axonal growth in iPSC sensory neurons, with in vivo rescue.
Anatomic Senso-DM™ enabled patient sensory neuron modeling.
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AAV9‐DARS2 Gene Therapy Rescues Phenotype in Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation Patient Cells and Neuronal Dars2 Deficient Mice
Objective
Leukoencephalopathy with brainstem and spinal cord involvement and lactate elevation (LBSL) is a rare, autosomal recessive disorder caused by variants in the gene DARS2. DARS2 is an essent.....