Disease correction in mucopolysaccharidosis type IIIB mice by intraparenchymal or cisternal delivery of a capsid modified AAV8 codon-optimized <i>NAGLU</i> vector
2022 · Human Molecular Genetics · 9 citations
https://doi.org/10.1093/hmg/ddac209
Z
Zackary M. Herbst
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Zackary M. Herbst is an academic researcher from Virginia Mason Medical Center. The author has contributed to research in topics: Lysosomal Storage Disorders Research & Trypanosoma species research and implications & Carbohydrate Chemistry and Synthesis. The author has an h-index of 11, co-authored 35 publications.
ORCID: 0000-0002-0097-4222Papers by this author
worksBiochemical signatures of disease severity in multiple sulfatase deficiency
2023 · Journal of Inherited Metabolic Disease · 7 citations
https://doi.org/10.1002/jimd.12688
Biochemical diagnosis of aromatic-L-amino acid decarboxylase deficiency (AADCD) by assay of AADC activity in plasma using liquid chromatography/tandem mass spectrometry
2022 · Molecular Genetics and Metabolism Reports · 6 citations
https://doi.org/10.1016/j.ymgmr.2022.100888
Corrigendum to “Evaluation of 3-O-methyldopa as a biomarker for aromatic L-amino acid decarboxylase deficiency in 7 Brazilian cases” [27/100744/2021/ pages: 1-4]
2022 · Molecular Genetics and Metabolism Reports · 1 citations
https://doi.org/10.1016/j.ymgmr.2022.100943
Corrigendum to “ Biochemical diagnosis of aromatic-L-amino acid decarboxylase deficiency (AADCD) by assay of AADC activity in plasma using liquid chromatography/tandem mass spectrometry” [32/100888 (2022) page 1–4].
2022 · Molecular Genetics and Metabolism Reports · 1 citations
https://doi.org/10.1016/j.ymgmr.2022.100945
High precision newborn screening for mucopolysaccharidosis type I by enzymatic activity followed by endogenous, non-reducing end glycosaminoglycan analysis
2024 · Molecular Genetics and Metabolism · 1 citations
https://doi.org/10.1016/j.ymgme.2024.108612
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