Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Rescue of a Mouse Model of Spinal Muscular Atrophy With Respiratory Distress Type 1 by AAV9-IGHMBP2 Is Dose Dependent

Molecular Therapy 24, 855 (May 2016). doi:10.1038/mt.2016.33

Authors: Monir Shababi, Zhihua Feng, Eric Villalon, Christine M Sibigtroth, Erkan Y Osman, Madeline R Miller, Patricka A Williams-Simon, Abby Lombardi, Thalia H Sass, Arleigh K Atkinson, Michael L Garcia, Chien-Ping Ko & Christian L Lorson

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