Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy.
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| Title: | Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy. |
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| Authors: | Mei C; Department of Pathophysiology and Transplantation (DEPT), Università degli studi di Milano, Milan, Italy.; Molecular Genetics and Functional Genomics, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy., Magliocca V; Molecular Genetics and Functional Genomics, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy., Chen X; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, United States., Massey K; Cure RTD Foundation, Calgary, AB, Canada., Gonzalez-Cordero A; Stem Cell Medicine Group, Children's Medical Research Institute, University of Sydney, Westmead, NSW, Australia., Gray SJ; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, United States., Tartaglia M; Molecular Genetics and Functional Genomics, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy., Bertini ES; Unit of Neuromuscular and Neurodegenerative Disorders, Translational Pediatrics and Clinical Genetics, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy., Corti S; Department of Pathophysiology and Transplantation (DEPT), Università degli studi di Milano, Milan, Italy., Compagnucci C; Molecular Genetics and Functional Genomics, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy. |
| Source: | Frontiers in cellular neuroscience [Front Cell Neurosci] 2025 Mar 11; Vol. 19, pp. 1523773. Date of Electronic Publication: 2025 Mar 11 (Print Publication: 2025). |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Frontiers Research Foundation Country of Publication: Switzerland NLM ID: 101477935 Publication Model: eCollection Cited Medium: Print ISSN: 1662-5102 (Print) Linking ISSN: 16625102 NLM ISO Abbreviation: Front Cell Neurosci Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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