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Last Updated: 10/31/2025
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Found 106 publications
Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA.
Journal: International journal of molecular sciences
Published: June 05, 2025
Recent advances in mucopolysaccharidosis IVA treatment.
Journal: Orphanet journal of rare diseases
Published: June 05, 2025
Case Report: Prenatal Diagnosis of Mucopolysaccharidosis IVA With Slow Growth of Long Bones: Identification of Novel Mutations in the GALNS Gene.
Journal: Congenital anomalies
Published: May 21, 2025
CRISPR/nCas9-Edited CD34+ Cells Rescue Mucopolysaccharidosis IVA Fibroblasts Phenotype.
Journal: International journal of molecular sciences
Published: March 19, 2025
Three-dimensional human mucopolysaccharidosis IVA chondrocyte culture reveals significant impairments in the lysosomal-mitochondrial crosstalk.
Journal: Scientific reports
Published: January 14, 2025
In vivo direct lentiviral gene therapy improves disease pathology in a mucopolysaccharidosis IVA murine model.
Journal: Molecular therapy. Methods & clinical development
Published: November 30, 2024
Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome).
Journal: Molecular genetics and metabolism reports
Published: September 25, 2024
Diminished GALNS activity in induced pluripotent stem cells of mucopolysaccharidosis IVA caused by compound p. S162Y and p. C165F mutation.
Journal: QJM : monthly journal of the Association of Physicians
Published: July 01, 2024
Diminished GALNS activity in induced pluripotent stem cells of mucopolysaccharidosis IVA caused by compound p.S162Y and p.C165F mutation.
Journal: QJM : monthly journal of the Association of Physicians
Published: July 01, 2024
Hematopoietic stem cell transplantation in children with mucopolysaccharidosis IVA: single center experience.
Journal: Bone marrow transplantation
Published: May 12, 2024
Last Updated: 10/31/2025