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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Positive early findings from ProQR trial of USH2A therapy
Biotechnology company ProQR has announced encouraging results from its early analysis of the phase 1/2 trial of QR-421a, an innovative approach to treating sight loss caused by mutations in a particular section of the USH2A gene.
CRISPR Gene Editing Leads to Improvements
CRISPR gene editing leads to improvements in vision for people with inherited blindness, clinical trial shows.
Drugs for cancer and alcoholism show promise for RP
With all the interest in exciting gene and cell therapies for inherited sight loss, it’s easy to forget that more traditional drugs can also hold promise.
New mechanism of disease discovered for Retinitis Pigmentosa
The team have found the cause of disease in the first ever family tree drawn up at Moorfields Eye Hospital over 35 years ago, which had remained unsolved until now.
Sirius clinical trial – Exon 13 mutation of USH2A
Prof Mariya Moosajee at Moorfields Eye Hospital has asked us to share the message below about the clinical trial she is running. This trial is for a treatment that targets a particular section of the USH2A gene.
Trusts and Foundations
Inherited retinal dystrophies (IRDs) are the leading cause of blindness in working-age people in the UK, and children as young as eighteen-months are regularly diagnosed.
Gene therapy shows promise in tackling X-linked RP
Early results from clinical testing of a gene therapy to treat X-linked retinitis pigmentosa (XLRP) have shown partial reversal of sight loss in some patients.
Webinar: How your donations make a difference…in conversation with our new research grantees
Join Research Development Manager, Kate Arkell as she speaks to our latest research grantees about their projects.
A new approach to treating inherited sight loss
A new approach to treating inherited sight loss