New Gene Therapy Offers Hope for Restoring Vision in Retinitis Pigmentosa Patients
Translated & summarized from Channel 13 by baba
A new gene therapy delivered by a single eye injection is offering hope to patients with retinitis pigmentosa, a condition causing progressive blindness. The treatment, developed by Professor Eitan Friedman at Assuta, creates a new protein in active cells rather than replacing a faulty gene. An early trial showed six of ten patients quadrupled their ability to recognize figures, a significant improvement for daily independence.
The story in 5 lines · by baba
- A new gene therapy offers hope for retinitis pigmentosa patients, potentially restoring lost vision.
- The treatment involves a single injection into the eye and creates a new protein in active cells.
- Six out of ten patients in an initial trial quadrupled their ability to recognize figures.
- Professor Eitan Friedman described the improvement as a significant step towards regaining independence.
- Retinitis pigmentosa is a rare, degenerative retinal disease leading to progressive blindness.
A novel gene therapy, administered via a single injection into the eye, is showing significant promise for individuals suffering from retinitis pigmentosa, a degenerative retinal disease previously considered untreatable. This new approach offers hope for restoring some lost vision.
Developed to combat the rare and severe condition that leads to progressive blindness, the therapy employs a unique mechanism. Professor Eitan Friedman, Director of the Genetic Institute for Early Detection at Assuta, explained in an interview that unlike traditional gene therapy, this treatment doesn't replace the faulty gene. Instead, it stimulates the creation of a new protein within still-active cells, a completely novel concept.
An initial clinical trial involving ten patients focused on the treatment's safety and its effect on unaffected nerve cells, aiming to assess improvements in visual function. The preliminary data revealed encouraging results regarding the practical impact on the daily lives of those with advanced vision loss. Specifically, six out of the ten participants demonstrated a quadrupled ability to recognize figures.
Professor Friedman highlighted the profound significance of this improvement, stating, "For a person dealing with severe vision loss, an improvement of this magnitude in recognition ability represents a significant turning point in their capacity to navigate their surroundings and perceive what is happening nearby, a first practical step towards regaining independence."
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