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Pre-clinical Antisense Oligonucleotide Treatment of CMT2E in a Human Induced Pluripotent Stem Cell (iPSC)-derived Motor Neuron Model

Author:Medina, Jessica   Rebelo, Adriana   Jacobs, Elizabeth   Danzi, Matt   Zuchner, Stephan   Saporta, Mario   

Session Name:S5: ALS and CMT: New Therapeutic Approaches  

Topic:Neuromuscular and Clinical Neurophysiology (EMG)  

Program Number:S5.008  

Author Institution:University of Miami, MIAMI, FL  University of Miami School of Medicine, Miami, FL  

Mechanisms of Neurodegeneration and Mini-gene Therapeutic Approach for Charcot-Marie-Tooth Disease Type 4B3

Author:Jacobs, Elizabeth   Danzi, Matt   Rebelo, Adriana   Zuchner, Stephan   Saporta, Mario   

Session Name:S37: Emerging Therapies in Child Neurology  

Topic:Child Neurology and Developmental Neurology  

Program Number:S37.007  

Author Institution:University of Miami Miller School of Medicine, Miami, FL