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Molecular Analysis of Vietnamese Patients with Mucopolysaccharidosis Type I

Life (Basel, Switzerland), 2021-10, Vol.11 (11), p.1162

2021

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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Molecular therapy, 2019-01, Vol.27 (1), p.178-187

2019

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Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Frontiers in molecular neuroscience, 2021-05, Vol.14, p.618360-618360

2021

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Mucopolysaccharidosis type I: founder effect of the p.P533R mutation in North Africa

BMC genomics, 2024-10, Vol.25 (1), p.948-9, Article 948

2024

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In silico analysis of potential off-target sites to gene editing for Mucopolysaccharidosis type I using the CRISPR/Cas9 system: Implications for population-specific treatments

PloS one, 2022-01, Vol.17 (1), p.e0262299-e0262299

2022

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Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Gene therapy, 2020-02, Vol.27 (1-2), p.74-84

2020

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Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system

The journal of gene medicine, 2022-04, Vol.24 (4), p.e3410-n/a

2022

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Open issues in Mucopolysaccharidosis type I-Hurler

Orphanet journal of rare diseases, 2017-06, Vol.12 (1), p.112-112, Article 112

2017

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Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan

Orphanet journal of rare diseases, 2018-05, Vol.13 (1), p.84-84, Article 84

2018

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Synthesis and Evaluation of Novel Triaryl Derivatives with Readthrough-Inducing Activity

Chemical and Pharmaceutical Bulletin, 2023/09/01, Vol.71(9), pp.701-716

2023

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MPSI Manifestations and Treatment Outcome: Skeletal Focus

International journal of molecular sciences, 2022-10, Vol.23 (19), p.11168

2022

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Mucopolysaccharidoses type I gene therapy

Journal of inherited metabolic disease, 2021-09, Vol.44 (5), p.1088-1098

2021

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An Integrated Computational Framework to Assess the Mutational Landscape of α‐L‐Iduronidase IDUA Gene

Journal of cellular biochemistry, 2018-01, Vol.119 (1), p.555-565

2018

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Estimated birth prevalence of mucopolysaccharidoses in Brazil

American journal of medical genetics. Part A, 2020-03, Vol.182 (3), p.469-483

2020

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Molecular docking analysis of a dermatan sulfate tetra-saccharide to human alpha-L-iduronidase

Bioinformation, 2023-12, Vol.19 (12), p.1116-1123

2023

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Why SNP rs3755955 is associated with human bone mineral density? A molecular and cellular study in bone cells

Molecular and cellular biochemistry, 2022-02, Vol.477 (2), p.455-468

2022

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IDUA gene mutations in mucopolysaccharidosis type‐1 patients from two Pakistani inbred families

Congenital anomalies, 2020-07, Vol.60 (4), p.126-127

2020

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Drosophila D-idua Reduction Mimics Mucopolysaccharidosis Type I Disease-Related Phenotypes

Cells (Basel, Switzerland), 2021-12, Vol.11 (1), p.129

2021

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Mucopolysaccharidosis Type I in the Russian Federation and Other Republics of the Former Soviet Union: Molecular Genetic Analysis and Epidemiology

Frontiers in molecular biosciences, 2022-01, Vol.8, p.783644-783644

2022

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p.X654R IDUA variant among Thai individuals with intermediate mucopolysaccharidosis type I and its residual activity as demonstrated in COS‐7 cells

Annals of human genetics, 2018-05, Vol.82 (3), p.150-157

2018

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Mapping of IDUA gene variants in Pakistani patients with mucopolysaccharidosis type 1

Journal of Pediatric Endocrinology and Metabolism, 2019-11, Vol.32 (11), p.1221-1227

2019

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IDUA Gene Variants and Response to Zoledronic Acid Treatment in Chinese Women with Postmenopausal Osteoporosis

Pharmacogenomics and personalized medicine, 2021-01, Vol.14, p.859-866

2021

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Sexual behaviour in a murine model of mucopolysaccharidosis type I (MPS I)

PloS one, 2019-12, Vol.14 (12), p.e0220429

2019

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Cardiac pathology in mucopolysaccharidosis I mice: Losartan modifies ERK1/2 activation during cardiac remodeling

Journal of inherited metabolic disease, 2021-05, Vol.44 (3), p.740-750

2021

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Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model

Stem cells international, 2019-01, Vol.2019 (2019), p.1-10

2019

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Molecular Analysis of Vietnamese Patients with Mucopolysaccharidosis Type I

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I

Mucopolysaccharidosis type I: founder effect of the p.P533R mutation in North Africa

In silico analysis of potential off-target sites to gene editing for Mucopolysaccharidosis type I using the CRISPR/Cas9 system: Implications for population-specific treatments

Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice

Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system

Open issues in Mucopolysaccharidosis type I-Hurler

Status of newborn screening and follow up investigations for Mucopolysaccharidoses I and II in Taiwan

Synthesis and Evaluation of Novel Triaryl Derivatives with Readthrough-Inducing Activity

MPSI Manifestations and Treatment Outcome: Skeletal Focus

Mucopolysaccharidoses type I gene therapy

An Integrated Computational Framework to Assess the Mutational Landscape of α‐L‐Iduronidase IDUA Gene

Estimated birth prevalence of mucopolysaccharidoses in Brazil

Molecular docking analysis of a dermatan sulfate tetra-saccharide to human alpha-L-iduronidase

Why SNP rs3755955 is associated with human bone mineral density? A molecular and cellular study in bone cells

IDUA gene mutations in mucopolysaccharidosis type‐1 patients from two Pakistani inbred families

Drosophila D-idua Reduction Mimics Mucopolysaccharidosis Type I Disease-Related Phenotypes

Mucopolysaccharidosis Type I in the Russian Federation and Other Republics of the Former Soviet Union: Molecular Genetic Analysis and Epidemiology

p.X654R IDUA variant among Thai individuals with intermediate mucopolysaccharidosis type I and its residual activity as demonstrated in COS‐7 cells

Mapping of IDUA gene variants in Pakistani patients with mucopolysaccharidosis type 1

IDUA Gene Variants and Response to Zoledronic Acid Treatment in Chinese Women with Postmenopausal Osteoporosis

Sexual behaviour in a murine model of mucopolysaccharidosis type I (MPS I)

Cardiac pathology in mucopolysaccharidosis I mice: Losartan modifies ERK1/2 activation during cardiac remodeling

Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model

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