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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.
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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PubType: Academic Journal
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  Data: Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
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  Data: <searchLink fieldCode="AU" term="%22Cain+JT%22">Cain JT</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.<br /><searchLink fieldCode="AU" term="%22Likhite+S%22">Likhite S</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22White+KA%22">White KA</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.<br /><searchLink fieldCode="AU" term="%22Timm+DJ%22">Timm DJ</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.<br /><searchLink fieldCode="AU" term="%22Davis+SS%22">Davis SS</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.<br /><searchLink fieldCode="AU" term="%22Johnson+TB%22">Johnson TB</searchLink>; Pediatrics and Rare Diseases Group, Sanford Research, Sioux Falls, SD 57104, USA.<br /><searchLink fieldCode="AU" term="%22Dennys-Rivers+CN%22">Dennys-Rivers CN</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Rinaldi+F%22">Rinaldi F</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Motti+D%22">Motti D</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Corcoran+S%22">Corcoran S</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Morales+P%22">Morales P</searchLink>; The Mannheimer Foundation, Inc., Homestead, FL 33034, USA.<br /><searchLink fieldCode="AU" term="%22Pierson+C%22">Pierson C</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Hughes+SM%22">Hughes SM</searchLink>; Department of Biochemistry, School of Biomedical Sciences, Brain Health Research Centre, University of Otago, Dunedin 9016, New Zealand.<br /><searchLink fieldCode="AU" term="%22Lee+SY%22">Lee SY</searchLink>; Division of Biology, Kansas State University, Manhattan, KS 66506, USA.<br /><searchLink fieldCode="AU" term="%22Kaspar+BK%22">Kaspar BK</searchLink>; AveXis Inc., Bannockburn, IL 60015, USA.<br /><searchLink fieldCode="AU" term="%22Meyer+K%22">Meyer K</searchLink>; The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, USA.<br /><searchLink fieldCode="AU" term="%22Weimer+JM%22">Weimer JM</searchLink>; 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.
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  Data: <searchLink fieldCode="JN" term="%22100890581%22">Molecular therapy : the journal of the American Society of Gene Therapy</searchLink> [Mol Ther] 2019 Oct 02; Vol. 27 (10), pp. 1836-1847. <i>Date of Electronic Publication: </i>2019 Jul 10.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>100890581 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1525-0024 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2215250016%22">15250016 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther <i>Subsets: </i>MEDLINE
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              Text: 2019 Oct 02
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