Establishment of mouse model of inherited PIGO deficiency and therapeutic potential of AAV-based gene therapy - Kuwayama, Ryoko; Suzuki, Keiichiro; Nakamura, Jun; Aizawa, Emi; Yoshioka, Yoshichika; Ikawa, Masahito; Nabatame, Shin; Inoue, Ken-ichi; Shimmyo, Yoshiari; Ozono, Keiichi; Kinoshita, Taroh; Murakami, Yoshiko (Nature communications, 2022)
Keywords: 13/109; 13/31; 13/51; 14/19; 14/63; 38/77; 42; 42/41; 42/44; 45; 45/88; 631/208/2489/201/2110; 64; 692/308/1426; 692/699/375; Animal models; Animals; Biosynthesis; CRISPR; Disease; Disease Models, Animal; Gene therapy; Genetic Therapy; Genomes; Glycosylphosphatidylinositol; Glycosylphosphatidylinositols - deficiency; Glycosylphosphatidylinositols - genetics; Homology; Humanities and Social Sciences; Immunoglobulin D; Immunoglobulin D - genetics; Mice; multidisciplinary; Mutation; Neurosciences; Phenotypes; Proteins; Research centers; Science; Science (multidisciplinary); Seizures - genetics