Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
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| Title: | Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease. |
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| Authors: | 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. |
| Source: | 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. |
| Publication Type: | Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't |
| Journal Info: | Publisher: Cell Press Country of Publication: United States NLM ID: 100890581 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1525-0024 (Electronic) Linking ISSN: 15250016 NLM ISO Abbreviation: Mol Ther Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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