Belite Bio – Phase 2/3 Trial for Adolescents with Stargardt disease
The latest research news piece from Retina UK.
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AAVantgarde Completes Enrolment in Phase 1/2 Clinical Trial for Usher Syndrome Type B
The latest research news piece from Retina UK.
x-linked inheritance
X-linked inheritance means that the faulty gene is located on the X chromosome, the larger of the two sex chromosomes.
The molecular map of congenital stationary night blindness – the science of seeing in the dark
A recently published study has provided the first molecular map of the mutation-specific changes that occur in the retina which lead to Congenital Stationary Night Blindness.
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.
Non-coding, but not non-important: the hidden genes behind retinitis pigmentosa
A new study by researchers from Radboud University Medical Centre and the University of Basel has uncovered new genetic causes of Retinitis Pigmentosa (RP) that were once overlooked.
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.
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.
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.
Research news round-up – Spring 2024
A round-up of the latest research into inherited retinal conditions - February 2024.