Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis.

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Title: Preclinical Enzyme Replacement Therapy with a Recombinant β-Galactosidase-Lectin Fusion for CNS Delivery and Treatment of GM1-Gangliosidosis.
Authors: Weesner JA; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.; Department of Anatomy and Physiology, University of Tennessee Health Science Center, Memphis, TN 38163, USA., Annunziata I; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.; Compliance Office, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Yang T; BioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA., Acosta W; BioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA., Gomero E; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Hu H; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., van de Vlekkert D; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Ayala J; BioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA., Qiu X; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Fremuth LE; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Radin DN; BioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA., Cramer CL; BioStrategies, LC, P.O. Box 2428, State University, Jonesboro, AR 72467, USA., d'Azzo A; Department of Genetics, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.; Department of Anatomy and Physiology, University of Tennessee Health Science Center, Memphis, TN 38163, USA.
Source: Cells [Cells] 2022 Aug 19; Vol. 11 (16). Date of Electronic Publication: 2022 Aug 19.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
Journal Info: Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409 (Electronic) Linking ISSN: 20734409 NLM ISO Abbreviation: Cells Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2073-4409
DOI:10.3390/cells11162579