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

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Title: Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease.
Authors: 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.
Source: Frontiers in genetics [Front Genet] 2023 Mar 24; Vol. 14, pp. 1118649. Date of Electronic Publication: 2023 Mar 24 (Print Publication: 2023).
Publication Type: Journal Article
Journal Info: Publisher: Frontiers Research Foundation Country of Publication: Switzerland NLM ID: 101560621 Publication Model: eCollection Cited Medium: Print ISSN: 1664-8021 (Print) Linking ISSN: 16648021 NLM ISO Abbreviation: Front Genet Subsets: PubMed not MEDLINE
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