Liver-directed AAV gene therapy in mice corrects glycogen storage disease type IX γ2.
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| Title: | Liver-directed AAV gene therapy in mice corrects glycogen storage disease type IX γ2. |
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| Authors: | Gibson RA; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27705, USA.; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Jeck WR; Department of Pathology, Duke University Medical Center, Durham, NC 27705, USA., Koch RL; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Jumani N; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Choi SJ; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Bali D; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Young SP; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Asokan A; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27705, USA.; Department of Surgery, Duke University Medical Center, Durham, NC 27705, USA., Lim JA; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA., Kishnani PS; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC 27705, USA.; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27705, USA. |
| Source: | Science advances [Sci Adv] 2025 Nov 14; Vol. 11 (46), pp. eady6760. Date of Electronic Publication: 2025 Nov 12. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101653440 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2375-2548 (Electronic) Linking ISSN: 23752548 NLM ISO Abbreviation: Sci Adv Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
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