Works matching IS 01418955 AND DT 2024 AND VI 47 AND IP 1
Results: 17
Liver‐directed gene therapy for inherited metabolic diseases.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 9, doi. 10.1002/jimd.12709
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Mission possible: Gene therapy for inherited metabolic diseases.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 5, doi. 10.1002/jimd.12708
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Whole genome sequencing to screen 100 000 newborns for treatable genetic disorders.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 7, doi. 10.1002/jimd.12704
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Gene therapy for mitochondrial disorders.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 145, doi. 10.1002/jimd.12699
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Gene therapy for neurotransmitter‐related disorders.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 176, doi. 10.1002/jimd.12697
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Correction to Maternal and fetal outcomes in acute hepatic porphyria: A Swedish National Cohort Study.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 212, doi. 10.1002/jimd.12685
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- Article
Correction to "Clinical presentation of 13 children with alkaptonuria".
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 211, doi. 10.1002/jimd.12683
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- Article
Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 63, doi. 10.1002/jimd.12665
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Fetal gene therapy.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 192, doi. 10.1002/jimd.12659
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Gene therapy for glycogen storage diseases.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 93, doi. 10.1002/jimd.12654
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State‐of‐the‐art 2023 on gene therapy for phenylketonuria.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 80, doi. 10.1002/jimd.12651
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Liver directed adeno‐associated viral vectors to treat metabolic disease.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 22, doi. 10.1002/jimd.12637
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Issue Information.
- Published in:
- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 1, doi. 10.1002/jimd.12627
- Publication type:
- Article
Gene therapies for mucopolysaccharidoses.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 135, doi. 10.1002/jimd.12626
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Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa<sup>−/−</sup> mice.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 119, doi. 10.1002/jimd.12625
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Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 50, doi. 10.1002/jimd.12609
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Successful treatment of severe MSUD in Bckdhb<sup>−/−</sup> mice with neonatal AAV gene therapy.
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- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 41, doi. 10.1002/jimd.12604
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- Article