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

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Apr 2018

Laoharawee Kanut, DeKelver Russell C, Podetz-Pedersen Kelly M, Rohde Michelle, Sproul Scott, Nguyen Hoang-Oanh, Nguyen Tam, St Martin Susan J, Ou Li, Tom Susan, Radeke Robert, Meyer Kathleen E, Holmes Michael C, Whitley Chester B, Wechsler Thomas, McIvor R Scott

Abstract excerpt

Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal disorder caused by deficiency of iduronate 2-sulfatase (IDS), leading to accumulation of glycosaminoglycans (GAGs) in tissues of affected individuals, progressive disease, and shortened lifespan. Currently available enzyme replacement therapy (ERT) requires lifelong infusions and does not provide neurologic benefit. We utilized a zinc...

Read the complete abstract on PubMed

Topics

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

Share to Endonucleases
Share to Energy Metabolism

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Gene Editing

Share to Gene Transfer Techniques

Share to Hepatocytes

Share to Iduronate Sulfatase

Share to Mucopolysaccharidosis II

Share to Zinc Fingers

Source

Open in PubMed Open full text

Identifiers

PMID: 29580682
DOI: 10.1016/j.ymthe.2018.03.002
PMCID: PMC6080131

Genes

IDS

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.PMID 29580682Read ↗

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler SyndromeSemantic proximity Read ↗

Prevention of Neurocognitive Deficiency in Mucopolysaccharidosis Type II Mice by Central Nervous System–Directed, AAV9-Mediated Iduronate Sulfatase Gene TransferSemantic proximity Read ↗

A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type ISemantic proximity Read ↗

First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia BSemantic proximity Read ↗

Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy.Semantic proximity Read ↗

A novel mucopolysaccharidosis type II mouse model with an iduronate-2-sulfatase-P88L mutation.Semantic proximity Read ↗

Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.Semantic proximity Read ↗

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?Semantic proximity Read ↗

<i>In vivo</i> adenine base editing corrects newborn murine model of Hurler syndromeSemantic proximity Read ↗

Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanismsSemantic proximity Read ↗

Therapeutic Options for Mucopolysaccharidosis II (Hunter Disease).Semantic proximity Read ↗

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type ISemantic proximity Read ↗

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