Please provide your email address to receive an email when new articles are posted on . The loss of UBAP1L gene function appears to be associated with nonsyndromic retinal dystrophy, according to ...

MeiraGTx Holdings plc announced promising results from a first-in-human study published in The Lancet, involving a gene therapy treatment for children with AIPL1-associated retinal dystrophy, ...

Scientists at the National Institutes of Health (NIH) and their colleagues have identified a gene responsible for some inherited retinal diseases (IRDs), which are a group of disorders that damage the ...

This story is part of a series on the current progression in Regenerative Medicine. This piece is part of a series dedicated to the eye and improvements in restoring vision. In 1999, I defined ...

Opus Genetics is conducting a trial for OPGx-MERTK gene therapy in Abu Dhabi, targeting MERTK-related retinitis pigmentosa ...

LONDON and NEW YORK, Feb. 21, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage genetic medicines company, today announced the publication of ...

ATLANTA, May 5, 2025 /PRNewswire/ -- Odylia Therapeutics, a nonprofit (501(c)3) biotechnology company dedicated to advancing treatments for rare diseases, today announced its latest pipeline project: ...

Please provide your email address to receive an email when new articles are posted on . BARCELONA, Spain — In this Healio Video Perspective from the Euretina congress, Bart Leroy, MD, PhD, speaks ...

Scientists in the UK have successfully used gene therapy to restore some vision to legally blind children with an inherited retinal condition. All 11 children in the clinical trial saw improvements ...

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 ...

Meaningful responses have been observed in 11 out of 11 LCA4 children treated to date with rAAV8.hRKp.AIPL1 All 11 children treated with rAAV8.hRKp.AIPL1 between 1 and 4 years old were legally blind ...