Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

9 Sept 2021

Abstract excerpt

Mucopolysaccharidosis type IVA (MPSIVA) or Morquio A disease, a lysosomal storage disorder, is caused by N-acetylgalactosamine-6-sulfate sulfatase (GALNS) deficiency, resulting in keratan sulfate (KS) and chondroitin-6-sulfate accumulation. Patients develop severe skeletal dysplasia, early cartilage deterioration and life-threatening heart and tracheal complications. There is no cure and enzyme replacement...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Lysosomal Storage Disorders Research
Share to Research on Leishmaniasis Studies
Share to Trypanosoma species research and implications

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 34504088
DOI: 10.1038/s41467-021-25697-y

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapyPMID 34504088Read ↗

Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVASemantic proximity Read ↗

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

Mucopolysaccharidosis IVA: Current Disease Models and DrawbacksSemantic proximity Read ↗

Liver-Targeted AAV8 Gene Therapy Ameliorates Skeletal and Cardiovascular Pathology in a Mucopolysaccharidosis IVA Murine ModelSemantic proximity Read ↗

Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal DiseasesSemantic proximity Read ↗

Molecular genetics and metabolism, special edition: Diagnosis, diagnosis and prognosis of Mucopolysaccharidosis IVA.Semantic proximity Read ↗

Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology.Semantic proximity Read ↗

Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Semantic proximity Read ↗

Suppression of Heterotopic Ossification in Fibrodysplasia Ossificans Progressiva Using AAV Gene DeliverySemantic proximity Read ↗

Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling ReactivationSemantic proximity Read ↗

Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA.Semantic proximity Read ↗

Treating neuromuscular diseases: unveiling gene therapy breakthroughs and pioneering future applicationsSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.