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Promising results for a first-in-class Usher syndrome IIA therapy

13/04/21 - Retinitis pigmentosa, Usher syndrome, Therapy,

The Dutch biotechnology company ProQR has just presented (March 2021) the first results of a clinical trial, named Stellar, with QR-421a, an antisense oligonucleotide designed to stop vision loss or restore vision in Usher syndrome IIA and non-syndromic retinitis pigmentosa patients due to USH2A exon 13 mutations. The therapeutical approach aims to exclude the exon 13 coding region in the...

Advances in gene therapy for retinitis pigmentosa due to RPGR gene mutation

12/05/20 - Retinal Dystrophy, Retinitis pigmentosa, Gene Therapy,

In the first months of 2020, positive results in gene therapy of X-linked retinitis pigmentosa (XLRP) have been reported. XLRP is an inherited genetic disorder that causes progressive vision loss, mainly in men. It begins in childhood with night blindness, progresses with the gradual reduction of the visual field and visual acuity, and leads to blindness in the fourth decade...