Rescue of a mouse model of spinal muscular atrophy with respiratory distress type 1 by AAV9-IGHMBP2 is dose dependent

Monir Shababi, Zhihua Feng, Eric Villalon, Christine Toedebusch, 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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