Mucopolysaccharidosis Type 4A (MPS IVA, Morquio Syndrome Type A) Latest Advances

Find the Latest Research About Mucopolysaccharidosis Type 4A (MPS IVA, Morquio Syndrome Type A)

Last Updated: 02/24/2026

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Found 109 publications

Long-Term Liver-Targeted AAV8 Gene Therapy for Mucopolysaccharidosis IVA.

Long-Term Liver-Targeted AAV8 Gene Therapy for Mucopolysaccharidosis IVA.

Journal: Current issues in molecular biology
Published: September 12, 2025

Early screening for respiratory and cardiac complications in pediatric mucopolysaccharidosis IVA: Insights from a case.

Early screening for respiratory and cardiac complications in pediatric mucopolysaccharidosis IVA: Insights from a case.

Journal: Intractable & rare diseases research
Published: September 01, 2025

Clinical and molecular spectrum of mucopolysaccharidosis IVA in Iraqi children: Allele-specific genotype-phenotype trends and novel GALNS variants.

Clinical and molecular spectrum of mucopolysaccharidosis IVA in Iraqi children: Allele-specific genotype-phenotype trends and novel GALNS variants.

Journal: Molecular genetics and metabolism reports
Published: July 11, 2025

Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA.

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.

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.

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.

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.

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.

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

Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice.

Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice.

Journal: Human gene therapy
Published: October 25, 2024

Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model.

Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model.

Journal: Human gene therapy
Published: October 24, 2024

Long-term outcomes of enzyme replacement therapy from a large cohort of Korean patients with mucopolysaccharidosis IVA (Morquio A syndrome).

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
Showing 1-12 of 109

Last Updated: 02/24/2026