首页> Orphanet journal of rare diseases> Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8<sup>mnd</sup> mice.

Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8<sup>mnd</sup> mice.

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第一作者： Andrew D,Holmes

第一单位： Pediatrics and Rare Diseases Group, Sanford Research, 2301 E 60Th St N, Sioux Falls, SD, USA.;Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD, USA.

作者： Andrew D,Holmes ^[1] ; Katherine A,White ^[2] ; Melissa A,Pratt ^[2] ; Tyler B,Johnson ^[2] ; Shibi,Likhite ^[3] ; Kathrin,Meyer ^[4] ; Jill M,Weimer ^[5]

作者单位： Pediatrics and Rare Diseases Group, Sanford Research, 2301 E 60Th St N, Sioux Falls, SD, USA.;Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD, USA. ^[1] Pediatrics and Rare Diseases Group, Sanford Research, 2301 E 60Th St N, Sioux Falls, SD, USA. ^[2] The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA. ^[3] The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.;Department of Pediatrics, The Ohio State University, Columbus, OH, USA. ^[4] Pediatrics and Rare Diseases Group, Sanford Research, 2301 E 60Th St N, Sioux Falls, SD, USA. Jill.Weimer@sanfordhealth.org.;Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD, USA. Jill.Weimer@sanfordhealth.org. ^[5]

关键词 AAV9 gene therapy Batten disease CLN8 Disease progression Lysosomal storage disorders Sex differences