Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.

Autor: Johnson TB; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States.; Amicus Therapeutics, Cranbury, NJ, United States., Brudvig JJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States.; Amicus Therapeutics, Cranbury, NJ, United States., Likhite S; The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States., Pratt MA; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States., White KA; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States., Cain JT; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States.; Amicus Therapeutics, Cranbury, NJ, United States., Booth CD; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States., Timm DJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States., Davis SS; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States., Meyerink B; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States., Pineda R; The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States., Dennys-Rivers C; The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States., Kaspar BK; The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States., Meyer K; The Research Institute at Nationwide Children's Hospital, Columbus, OH, United States., Weimer JM; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD, United States.; Amicus Therapeutics, Cranbury, NJ, United States.; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Vermillion, SD, United States.

Publikováno v: Frontiers in genetics [Front Genet] 2023 Mar 24; Vol. 14, pp. 1118649. Date of Electronic Publication: 2023 Mar 24 (Print Publication: 2023).

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.

Autor: Cain JT; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA., Likhite S; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., White KA; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA., Timm DJ; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA., Davis SS; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA., Johnson TB; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA., Dennys-Rivers CN; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Rinaldi F; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Motti D; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Corcoran S; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Morales P; The Mannheimer Foundation, Inc., Homestead, FL 33034, USA., Pierson C; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Hughes SM; Department of Biochemistry, School of Biomedical Sciences, Brain Health Research Centre, University of Otago, Dunedin 9016, New Zealand., Lee SY; Division of Biology, Kansas State University, Manhattan, KS 66506, USA., Kaspar BK; AveXis Inc., Bannockburn, IL 60015, USA., Meyer K; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA., Weimer JM; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA; Department of Pediatrics, Sanford School of Medicine, University of South Dakota, Sioux Falls, SD 57069, USA. Electronic address: jill.weimer@sanfordhealth.org.

Publikováno v: Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2019 Oct 02; Vol. 27 (10), pp. 1836-1847. Date of Electronic Publication: 2019 Jul 10.