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

Split AAV8 Mediated ABCA4 Expression for Gene Therapy of Mouse Stargardt Disease (STGD1).

Human gene therapy - 1 Jul 2023

Li Ruiting, Jing Qiuping, She Kaiqin, Wang Qingnan, Jin Xiu, Zhao Qinyu, Su Jing, Song Li, Fu Jiamei, Wu Xiaoyi, Xu Qiuxia, Lu Fang, Wei Yuquan, Yang Yang

Abstract excerpt

Adeno-associated virus (AAV)-based gene therapy has been shown to be safe and effective in numerous animal models and clinical trials for various ophthalmic diseases. Stargardt disease (STGD1; MIM #248200) is the most common autosomal recessive macular dystrophy disease, and the most common form is caused by mutations in the ABCA4 gene, a gene with 6.8 kb coding sequence. Split intein approaches increase the...

Read the complete abstract on PubMed

Topics

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

Share to Genetic Therapy
Share to Macular Degeneration

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mice, Knockout

Share to Mutation

Share to Retinal Diseases

Share to Stargardt Disease

Source

Identifiers

PMID: 37227014
DOI: 10.1089/hum.2023.017

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Split AAV8 Mediated ABCA4 Expression for Gene Therapy of Mouse Stargardt Disease (STGD1).PMID 37227014Read ↗

High-efficiency base editing for Stargardt disease in mice, non-human primates, and human retina tissueSemantic proximity Read ↗

Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retinaSemantic proximity Read ↗

Dual-AAV split prime editor corrects the mutation and phenotype in mice with inherited retinal degeneration.Semantic proximity Read ↗

Updates on Emerging Interventions for Autosomal Recessive ABCA4-Associated Stargardt DiseaseSemantic proximity Read ↗

The ABCs of Stargardt disease: the latest advances in precision medicineSemantic proximity Read ↗

Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review.Semantic proximity Read ↗

Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse model.Semantic proximity Read ↗

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapySemantic proximity Read ↗

Gene Therapy of ABCA4-Associated Diseases.Semantic proximity Read ↗

An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Abca4 −/− MiceSemantic proximity Read ↗

Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy.Semantic proximity Read ↗

Generation of two induced pluripotent stem cell lines from a patient with Stargardt disease caused by compound heterozygous mutations in the ABCA4 gene.Semantic proximity Read ↗

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