Last Updated: 09/26/2026
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Found 5 publications
The Ighmbp2D564N mouse model is the first SMARD1 model to demonstrate respiratory defects.
Journal: Human molecular genetics
Published: August 12, 2021
Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2 nmd-2J.
Journal: Molecular therapy. Methods & clinical development
Published: April 07, 2021
AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice.
Journal: Biochemical and biophysical research communications
Published: June 25, 2020
Muscle fiber-type selective propensity to pathology in the nmd mouse model of SMARD1.
Journal: Biochemical and biophysical research communications
Published: June 11, 2019
AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/-SMA model mice.
Journal: Human molecular genetics
Published: February 26, 2019
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Last Updated: 09/26/2026