Sgcb-flox 基因敲除小鼠

1. Mendell, Jerry R, Pozsgai, Eric R, Lewis, Sarah, Stevenson, Herb, Rodino-Klapac, Louise R. 2024. Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. In Nature medicine, 30, 199-206. doi:10.1038/s41591-023-02730-9. https://pubmed.ncbi.nlm.nih.gov/38177855/

2. Magri, Francesca, Zanotti, Simona, Salani, Sabrina, Comi, Giacomo Pietro, Ronchi, Dario. 2022. Antisense Morpholino-Based In Vitro Correction of a Pseudoexon-Generating Variant in the SGCB Gene. In International journal of molecular sciences, 23, . doi:10.3390/ijms23179817. https://pubmed.ncbi.nlm.nih.gov/36077211/

3. Vainzof, Mariz, Souza, Lucas S, Gurgel-Giannetti, Juliana, Zatz, Mayana. 2021. Sarcoglycanopathies: an update. In Neuromuscular disorders : NMD, 31, 1021-1027. doi:10.1016/j.nmd.2021.07.014. https://pubmed.ncbi.nlm.nih.gov/34404573/

4. Ten Dam, Leroy, Frankhuizen, Wendy S, Linssen, Wim H J P, van der Kooi, Anneke J, Ginjaar, Ieke. 2019. Autosomal recessive limb-girdle and Miyoshi muscular dystrophies in the Netherlands: The clinical and molecular spectrum of 244 patients. In Clinical genetics, 96, 126-133. doi:10.1111/cge.13544. https://pubmed.ncbi.nlm.nih.gov/30919934/