Retinitis pigmentosa (RP) is an inherited retinal dystrophy that causes loss of vision. Pathogenic variants in proteins involved in RNA splicing are the second most common cause of autosomal dominant RP, with mutations in PRPF31 being the most prevalent. Additionally, mutations in spliceosomal small nuclear RNAs (snRNAs) U4 and U6 have recently been linked to RP.
Opus Genetics Inc. is advancing a pipeline of gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases, with three programs expected to enter clinical testing over the next 12-18 months.
Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional CRISPR/Cas9 editing.
Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing.
Researchers at Carl von Ossietzky Universität Oldenburg generated retinal pigment epithelial cell lines lacking three RPGR isoforms and examined whether they showed the defects in ciliary structure and actin turnover known to occur in retinitis pigmentosa.
Axovia Therapeutics Inc. has been awarded a new $1.0 million grant by the nonprofit organization A Race Against Blindness to support the clinical development of AXV-101, an investigational gene therapy aimed at combating childhood blindness due to retinitis pigmentosa caused by Bardet-Biedl syndrome 1 (BBS1).
Aussie gene therapy company Mirugen Pty Ltd. has emerged from stealth mode from the Center for Eye Research Australia in Melbourne, toting a AU$4.5 million (US$2.9 million) seed round that will see it optimize its lead candidate to treat retinitis pigmentosa.
Aussie gene therapy company Mirugen Pty Ltd. has emerged from stealth mode from the Center for Eye Research Australia in Melbourne, toting a AU$4.5 million (US$2.9 million) seed round that will see it optimize its lead candidate to treat retinitis pigmentosa.
Variant SAS and the Rare Ocular Diseases Center at the University of Campania Luigi Vanvitelli (UCLV) have received positive feedback from the EMA for VAR-002, a recombinant AAV vector gene therapy targeting inherited retinal dystrophies linked to CRX mutations.
Inherited retinal dystrophies (IRDs), including retinitis pigmentosa and Stargardt disease, are a group of rare degenerative disorders of the retina with clinical and genetic heterogeneity. In a recent publication, researchers from the Institute of Ocular Microsurgery applied clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 and transcription activator-like effector nucleases (TALEN) gene-editing tools to precisely correct induced pluripotent stem cell (hiPSC) lines derived from IRD patients.
-and-Raymond-Wong-(R).webp?height=488&t=1755204408&width=650 "Mirugen co-founders Keith Martin (L) and Raymond Wong (R)")
