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

AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa.

International journal of molecular sciences - 2 Feb 2024

Sladen Paul E, Naeem Arifa, Adefila-Ideozu Toyin, Vermeule Tijmen, Busson Sophie L, Michaelides Michel, Naylor Stuart, Forbes Alexandria, Lane Amelia, Georgiadis Anastasios

Abstract excerpt

Variants within the Retinitis Pigmentosa GTPase regulator (RPGR) gene are the predominant cause of X-Linked Retinitis Pigmentosa (XLRP), a common and severe form of inherited retinal disease. XLRP is characterised by the progressive degeneration and loss of photoreceptors, leading to visual loss and, ultimately, bilateral blindness. Unfortunately, there are no effective approved treatments for RPGR-associated...

Read the complete abstract on PubMed

Topics

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

Share to Carrier Proteins
Share to Dependovirus

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Eye Proteins

Share to Genetic Therapy

Share to Opsins

Share to Reactive Oxygen Species

Share to Retinitis Pigmentosa

Share to Rhodopsin

Source

Open in PubMed Open full text

Identifiers

PMID: 38339118
DOI: 10.3390/ijms25031839
PMCID: PMC10855600

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis Pigmentosa.PMID 38339118Read ↗

Molecular Strategies for RPGR Gene TherapySemantic proximity Read ↗

Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa.Semantic proximity Read ↗

RPGR-associated retinal degeneration in human X-linked RP and a murine model.Semantic proximity Read ↗

Gene Correction Reverses Ciliopathy and Photoreceptor Loss in iPSC-Derived Retinal Organoids from Retinitis Pigmentosa Patients.Semantic proximity Read ↗

Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the HorizonSemantic proximity Read ↗

RPGR: Its role in photoreceptor physiology, human disease, and future therapiesSemantic proximity Read ↗

Single-Cell Transcriptomic Dataset of RPGR-associated Retinitis Pigmentosa Patient-Derived Retinal Organoids.Semantic proximity Read ↗

Knockout ofRP2decreases GRK1 and rod transducin subunits and leads to photoreceptor degeneration in zebrafishSemantic proximity Read ↗

RPGR-Related Retinopathy: Clinical Features, Molecular Genetics, and Gene Replacement Therapy.Semantic proximity Read ↗

Variegated yet non-random rod and cone photoreceptor disease patterns in RPGR-ORF15-associated retinal degeneration.Semantic proximity Read ↗

Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa.Semantic proximity Read ↗

Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing.Semantic proximity Read ↗

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