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

CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I

17 Dec 2018

Abstract excerpt

Abstract CRISPR/Cas9 technology offers novel approaches for the development of new therapies for many unmet clinical needs, including a significant number of inherited monogenic diseases. However, in vivo correction of disease-causing genes is still inefficient, especially for those diseases without selective advantage for corrected cells. We reasoned that substrate reduction therapies (SRT) targeting...

Read the complete abstract on PubMed

Topics

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

Share to Biomedical Research and Pathophysiology
Share to CRISPR and Genetic Engineering
Share to Organ Donation and Transplantation

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Source

Identifiers

PMID: 30575740
DOI: 10.1038/s41467-018-07827-1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type IPMID 30575740Read ↗

In vivo CRISPR-Cas9 inhibition of hepatic LDH as treatment of primary hyperoxaluriaSemantic proximity Read ↗

Knockdown of lactate dehydrogenase by adeno‐associated virus‐delivered CRISPR/Cas9 system alleviates primary hyperoxaluria type 1Semantic proximity Read ↗

Human glyoxylate metabolism revisited: New insights pointing to multi-organ involvement with implications for siRNA-based therapies in primary hyperoxaluria.Semantic proximity Read ↗

A molecular journey on the pathogenesis of primary hyperoxaluria.Semantic proximity Read ↗

Deep phenotyping of a healthy human HAO1 knockout informs therapeutic development for primary hyperoxaluria type 1Semantic proximity Read ↗

[From gene to disease; primary hyperoxaluria type I caused by mutations in the AGXT gene].Semantic proximity Read ↗

Primary hyperoxalurias: disorders of glyoxylate detoxification.Semantic proximity Read ↗

Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblastsSemantic proximity Read ↗

Primary hyperoxaluria: insights into its clinical presentation, genetic mutations, and transplantation outcomes in a pediatric population in a tertiary care center.Semantic proximity Read ↗

Primary HyperoxaluriaSemantic proximity Read ↗

Primary hyperoxaluria in Italy: the past 30 years and the near future of a (not so) rare disease.Semantic proximity Read ↗

Molecular etiology of primary hyperoxaluria type 1: new directions for treatment.Semantic proximity Read ↗

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