News

Precision CRISPR repairs blindness in mice

In genome-editing, the challenge for CRISPR-wielding scientists is to edit only one of the two copies, or alleles, of every gene that people have, repairing the ever-so-slightly broken one and …

Gene therapy company receives Rare Paediatric Disease Designation for the treatment of Achromatopsia

MeiraGTx, a London and New York-based gene therapy company, has announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have …

Blood-Retina Chip research

Researchers from Microelectronics Institute of Barcelona, Vall d’Hebron Research Institute, and Universitat Autònoma de Barcelona have developed a microfluidic chip that mimics the human blood-retinal barrier in the eye. The …

London opera singer with RP to appear as first blind female TV Judge

Lizzie Capener, a gifted opera singer will be the first blind female TV judge; she appears in the show, All Together Now, which has been described as a “singing contest …

Blind – A sixth Sense art exhibition

We’re pleased to share details of an exhibition that’s taking place in February showcasing the sculptures of a visually impaired artist. Victoria Claire is a Kent-based artist, specialising in contemporary …

GenSight to start clinical testing combination of gene therapy and wearable device

GenSight will start a clinical trial in the UK testing a combination of gene therapy and a wearable device to restore sight in patients with retinitis pigmentosa. The UK Medicines …

Agreement formed for development of AAV vectors for ocular gene therapy

Sirion Biotech GmbH, a virus-based technology and gene delivery company, announced that it has signed a two-year development agreement with Acucela, a clinical-stage ophthalmology company. Recombinant adeno associated virus (rAAV) …

RP Fighting Blindness Membership

We’d like to say a big thank you to everyone who plans to continue supporting RP Fighting Blindness in 2018 as a member. The membership subscriptions are a vital addition …

FDA approves Luxturna gene therapy treatment

The U.S. Food and Drug Administration has approved Luxturna (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may …

jCyte Presents Results of Clinical Testing in RP

Cell therapy company jCyte has announced results from a phase 1/2a clinical trial for its investigational product, jCell, in retinitis pigmentosa. Presented by David S. Boyer, MD, at the annual …

Scientists explore how a pain medicine also protects vision in blinding conditions

Scientists have shown that a medicine prescribed for severe pain can help preserve vision in a model of severe, blinding retinal degeneration. Now they want to know more about how …

ABCA4 midigenes reveal the full splice spectrum of all reported noncanonical splice site variants in Stargardt disease

Based on the work of Riccardo Sangermano, Mubeen Khan, Stephanie Cornelis, PhD candidates in the group of prof. Frans Cremers in the Department of Human Genetics, Radboudumc, Nijmegen, The Netherlands, …