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The Ighmbp2D564N mouse model is the first SMARD1 model to demonstrate respiratory defects.
Smith CE, Lorson MA, Ricardez Hernandez SM, Al Rawi Z, Mao J, Marquez J, Villalón E, Keilholz AN, Smith CL, Garro-Kacher MO, Morcos T, Davis DJ, Bryda EC, Nichols NL, Lorson CL. Smith CE, et al. Among authors: garro kacher mo. Hum Mol Genet. 2022 Apr 22;31(8):1293-1307. doi: 10.1093/hmg/ddab317. Hum Mol Genet. 2022. PMID: 34726235 Free PMC article.
Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2 nmd-2J.
Shababi M, Smith CE, Ricardez Hernandez SM, Marquez J, Al Rawi Z, Villalón E, Farris KD, Garro-Kacher MO, Lorson CL. Shababi M, et al. Among authors: garro kacher mo. Mol Ther Methods Clin Dev. 2021 Aug 8;23:23-32. doi: 10.1016/j.omtm.2021.07.008. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34553000 Free PMC article.