Original Research
Accepted on 27 Mar 2026
RNA Activation as a Precision Dosing Modality: MTL-CEBPA for Controlled Enzyme Elevation in MPS I-H
in Gene and Cell Therapy
Frontiers in Medicine
- 342 views
10 articles related to "Idua"
Articles
Original Research
Published on 15 Jul 2024
Enzymatic testing for mucopolysaccharidosis type I in Kuwaiti newborns: a preliminary study toward newborn screening
in Genetics of Common and Rare Diseases
Frontiers in Pediatrics
doi 10.3389/fped.2024.1376053
- 2,743 views
- 2 citations
Case Report
Published on 27 Feb 2026
Case Report: Compound heterozygous mutations in the IDUA gene causing mucopolysaccharidosis type I with uterine developmental abnormality
in Pediatric Endocrinology
Frontiers in Pediatrics
doi 10.3389/fped.2026.1768349
- 384 views
Original Research
Published on 10 May 2021
Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type I
in Molecular Neuroscience Archive
Frontiers in Molecular Neuroscience
doi 10.3389/fnmol.2021.618360
- 15,750 views
- 44 citations
Original Research
Published on 24 Jan 2022
Mucopolysaccharidosis Type I in the Russian Federation and Other Republics of the Former Soviet Union: Molecular Genetic Analysis and Epidemiology
in Molecular Diagnostics and Therapeutics
Frontiers in Molecular Biosciences
doi 10.3389/fmolb.2021.783644
- 8,964 views
- 8 citations
Case Report
Published on 16 Aug 2022
Treatment of latent tuberculosis in a child with mucopolysaccharidosis type I receiving enzyme replacement therapy: A case report
in Genetics of Common and Rare Diseases
Frontiers in Pediatrics
doi 10.3389/fped.2022.973193
- 2,331 views
Original Research
Published on 21 Oct 2021
Which Is the Best In Silico Program for the Missense Variations in IDUA Gene? A Comparison of 33 Programs Plus a Conservation Score and Evaluation of 586 Missense Variants
in Molecular Diagnostics and Therapeutics
Frontiers in Molecular Biosciences
doi 10.3389/fmolb.2021.752797
- 7,589 views
- 12 citations
Original Research
Published on 11 Dec 2023
Leveraging diverse cell-death related signature predicts the prognosis and immunotherapy response in renal clear cell carcinoma
in Cancer Immunity and Immunotherapy
Frontiers in Immunology
doi 10.3389/fimmu.2023.1293729
- 4,121 views
- 8 citations
Case Report
Published on 19 Jan 2024
Evaluation of etanercept (a tumor necrosis factor alpha inhibitor) as an effective treatment for joint disease in mucopolysaccharidosis type I. A case report with whole-body magnetic resonance imaging
in Rheumatology
Frontiers in Medicine
doi 10.3389/fmed.2023.1252704
- 2,329 views
- 3 citations
Original Research
Published on 28 Mar 2022
Establishment of Cutoff Values for Newborn Screening of Six Lysosomal Storage Disorders by Tandem Mass Spectrometry
in Genetics of Common and Rare Diseases
Frontiers in Pediatrics
doi 10.3389/fped.2022.814461
- 7,238 views
- 11 citations