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              In-vitro Efficacy and In-vivo Biodistribution Studies of an Antisense Oligonucleotide Targeting Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation

              Author:Amanat, Manouchehr   Nemeth, Christina   Garofolo, Ines   Fine, Amena   Fatemi, Ali   

              Session Name:S37: Emerging Therapies in Child Neurology  

              Topic:Child Neurology and Developmental Neurology  

              Program Number:S37.010  

              Author Institution:Johns Hopkins School of Medicine, Baltimore, MD  Kennedy Krieger Institute, Baltimore, MD