x-linked inheritance
X-linked inheritance means that the faulty gene is located on the X chromosome, the larger of the two sex chromosomes.
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Meet a researcher: Hassina Zeriri
It’s not very often that we come across a researcher working on a project inspired by their own lived experience of a sight loss condition.
Brain stimulation may reduce hallucinations
UK researchers have discovered that passing a weak electrical current between electrodes on a person’s scalp may lead to a reduction in frequency of the visual hallucinations experienced by some people living with sight loss.
Potential drug targets identified for RP
A recently published study has described numerous disease mechanisms that appear to be common across different types of inherited sight loss, suggesting that there is significant potential for drug treatments that could work regardless of the underlying causative gene.
Autosomal dominant inheritance
Autosomal dominant inheritance means that the faulty gene is on an autosome (any chromosome other than the sex chromosomes) and that only one faulty copy is needed to cause sight loss.
Gene Therapy improves functional measures in XLRP
Beacon Therapeutics announced positive results from their phase 2 gene therapy trial for X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
SB-007 update: dose-expansion portion of SpliceBio’s phase 1/2 trial underway with first participant dosed at Oxford
SpliceBio has begun the dose-expansion portion of their phase 1/2 clinical trial of SB-007, a dual adeno-associated viral vector gene therapy for Stargardt disease.
Significantly worse vision found in female carriers of X-linked RP and Choroideremia
It was previously believed that female carriers of X-linked inherited retinal diseases (IRDs) like X-linked retinitis pigmentosa (RP and Choroideremia) remained unaffected by sight loss.
Retina-on-a-chip: the tiny technology offering big hope for inherited sight loss
Researchers in the Netherlands are developing a “retina-on-a-chip” that mimics the key layers of the human retina, offering a powerful new way to study inherited retinal diseases (IRDs) and test potential treatments using human cells.
RTx-015: Exploring Optogenetic Gene Therapy for RP and Choroideremia
Ray Therapeutics, a clinical stage biopharmaceutical company, is currently investigating their lead optogenetic therapy candidate, RTx-015, in early stage clinical trials for those with RP and Choroideremia.