• Email Us: [email protected]
  • Contact Us: +1 718 874 1545
  • Skip to main content
  • Skip to primary sidebar

Medical Market Report

  • Home
  • All Reports
  • About Us
  • Contact Us

World-First Gene Therapy Improves Vision For Man With Rare, Previously Untreatable Form Of Blindness

August 4, 2025 by Deborah Bloomfield

A man born with Usher syndrome type 1b, a rare genetic disease that causes congenital deafness and progressive blindness, has reportedly experienced “substantial improvement” in his vision, after receiving a new type of gene therapy as part of an ongoing clinical trial.

The 38-year-old man was the first patient in the world to be given the new treatment, and a year later, he stands as the first evidence that it can work – and how life-changing that could be.

“Now I can recognize faces, see the warehouse aisles at work, and read subtitles on TV. It’s not just about seeing better – it’s about starting to live again,” said the patient in a statement.

Usher syndrome is caused by mutations in a gene called MYO7A, which provides the instructions to produce a protein that plays a critical role in the inner ear and the retina, which converts light into electrical signals that our brain decodes as images. 

The mutations in MYO7A mean that people with this condition are typically born with severe to total hearing loss, and progressive vision loss that usually begins as a teenager or in young adulthood.

Gene therapy for such conditions is a rapidly expanding field, but when it comes to Usher syndrome type 1b, there’s a problem – MYO7A is a big gene. Too big, in fact, to be carried by the viral vectors that are typically used to deliver healthy copies of a gene to cells in existing therapies.

So, researchers at the Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli, Italy, came up with a possible solution: split the gene in half. Instead of just one vector, the gene is spread over two. Once delivered, cells in the retina can reassemble the two halves and use them to produce a fully functional copy of the protein MYO7A encodes.

In theory, this should lead to restoration of vision. The initial findings from the trial, which has so far involved eight patients, suggest that seems to be the case in practice – and quickly, too.

 “In the first patient treated, improvements were evident just two weeks after the procedure and continued over time,” said Professor Francesca Simonelli, head of the Center for Advanced Ocular Therapies at Vanvitelli University, one of the centers taking part in the trial. These improvements were in both near and distant vision, and even when there was little light.

But it’s not just about whether a treatment works – it needs to be safe as well. Again, that appears to be the case here, with no serious adverse events reported, and the side effects that did arise being only mild, infrequent, and easily managed.

Still, it’s important to emphasize that these are preliminary findings, with the trial set to continue over the coming months by testing out a higher dose of the treatment in a further seven patients. Even if all goes well, there’s still a long road ahead before the therapy could be approved – but the researchers are feeling positive.

“This is still an ongoing trial, but the signals observed so far offer new hope for other inherited eye diseases that currently have no treatment options,” Simonelli concluded.

Deborah Bloomfield
Deborah Bloomfield

Related posts:

  1. Audi launches its newest EV, the 2022 Q4 e-tron SUV
  2. Dinosaur Prints Found Under Restaurant Table Confirmed As 100 Million Years Old
  3. Archax: Japanese Engineers Make Transformer Robot That Actually Works
  4. How Do We Know There Is Anything Beyond The Observable Universe?

Source Link: World-First Gene Therapy Improves Vision For Man With Rare, Previously Untreatable Form Of Blindness

Filed Under: News

Primary Sidebar

  • An Alien-Like Fish With A See-Through Head And Green Eyes Lurks In The Ocean’s Dark Depths
  • Africa Wants To Change Misleading World Map, The “Wow!” Signal Was Likely From An Extraterrestrial Source, And Much More This Week
  • A “Good Death”: How Do Doctors Want To Die?
  • People Are Throwing Baby Puffins Off Cliffs In Iceland Again – But Why?
  • Yet Another Ancient Human Skull Turns Out To Be Denisovan
  • Gen Z Might Not Be On Course For A Midlife Crisis – Good News, Right? Wrong
  • Glowing Plants, Punk Ankylosaur, And Has The Wow! Signal Been Solved?
  • Pulsar Fleeing A Supernova Spotted Where Neither Of Them Should Be
  • 20 Years After Hurricane Katrina: Is It Time For A New Approach To Hurricane Classification?
  • Dog Named Scribble Replicates Quantum Factorization Records – So We Tried It Too
  • How Old Is The Solar System? (And How Can We Tell?)
  • Next Week, A Record-Breaking Over 7 Billion People Will See The Total Lunar Eclipse
  • The Goblin Shark Has The Fastest Jaws In The Ocean, Firing Like A Slingshot At Speeds Of 3.1-Meters-Per-Second
  • We Thought Geological Boundaries Were Random. Now, A New Study Has Identified Hidden Patterns
  • Do Fish Sleep?
  • The Biblical Flood Myth That Inspired Noah’s Ark Had A Sinister Twist
  • Massive Review Of 19 Autism Therapies Finds No Strong Evidence And Lack Of Safety Data
  • Giant City-Swallowing Cracks In Earth’s Surface Are A “New Geo-Hydrological Hazard”
  • Three Incredible Telescopes Looked At The Butterfly Nebula To Learn Where Earth Came From
  • The Pacific Ocean Is So Vast It Contains Its Own Antipodes
  • Business
  • Health
  • News
  • Science
  • Technology
  • +1 718 874 1545
  • +91 78878 22626
  • [email protected]
Office Address
Prudour Pvt. Ltd. 420 Lexington Avenue Suite 300 New York City, NY 10170.

Powered by Prudour Network

Copyrights © 2025 · Medical Market Report. All Rights Reserved.

Go to mobile version