Fkrp-flox 基因敲除小鼠

1. Cataldi, Marcela P, Vannoy, Charles H, Blaeser, Anthony, Holbrook, Molly C, Lu, Qi Long. 2023. Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I. In Molecular therapy : the journal of the American Society of Gene Therapy, 31, 3478-3489. doi:10.1016/j.ymthe.2023.10.022. https://pubmed.ncbi.nlm.nih.gov/37919902/

2. Brown, Susan C, Fernandez-Fuente, Marta, Muntoni, Francesco, Vissing, John. . Phenotypic Spectrum of α-Dystroglycanopathies Associated With the c.919T>a Variant in the FKRP Gene in Humans and Mice. In Journal of neuropathology and experimental neurology, 79, 1257-1264. doi:10.1093/jnen/nlaa120. https://pubmed.ncbi.nlm.nih.gov/33051673/

3. Dhoke, Neha R, Kim, Hyunkee, Selvaraj, Sridhar, Bang, Anne G, Perlingeiro, Rita C R. . A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy. In Cell reports, 36, 109360. doi:10.1016/j.celrep.2021.109360. https://pubmed.ncbi.nlm.nih.gov/34260922/