Radical new gene therapy restores sight to patients with rare eye condition - BBC News
Ringkasan
TLDRThe report by Fergus Walsh on BBC News highlights a revolutionary gene therapy called Luxturna which is being used for patients with a rare inherited eye condition that causes severe tunnel vision. The therapy has shown unexpected benefits by not only halting the further loss of vision but also significantly improving eyesight in patients like Jake and Matthew who were losing their sight due to this condition. This involves an expensive treatment that injects working copies of the RPE 65 gene into the retina, encapsulated in a harmless virus. The NHS, collaborating with the manufacturer Novartis, has arranged a discounted price for the therapy. The therapy provides hope for those with similar conditions, especially children, as it might prevent permanent sight damage.
Takeaways
- š¬ Innovative gene therapy called Luxturna is helping patients with inherited eye conditions.
- š° The therapy, though costly, is covered by NHS at a discounted rate.
- šļø Jake, a patient, experienced improved peripheral vision and ability to see facial features.
- š¬ The therapy involves injecting a functional gene into the retina using a virus.
- šØāš©āš§ Children are likely to benefit the most as therapy can prevent permanent sight damage.
- š Patients like Matthew report increased independence due to regained vision abilities.
- š„ Treatment offered in specialized hospitals like Moorfield's Eye Hospital in London.
- š Therapy marks transformative change offering hope to patients with rare conditions.
- š” Experience slight visual improvement boosts patient confidence and daily life quality.
- š§¬ Novartis, the manufacturer, collaborates with healthcare providers for treatment accessibility.
Garis waktu
- 00:00:00 - 00:03:18
Jake has been experiencing gradual loss of vision due to a rare inherited eye condition that causes tunnel vision. However, a new form of gene therapy has not only halted further vision loss but also improved his vision. The gene therapy involves injecting a working copy of a faulty gene, RPE65, into the retina. This process is hoped to stabilize and improve vision further for Jake and others with similar conditions.
Peta Pikiran
Pertanyaan yang Sering Diajukan
What is the rare inherited eye condition mentioned?
It is a condition that causes severe tunnel vision due to a faulty RPE 65 gene.
What is the name of the gene therapy used in the report?
The gene therapy is called Luxturna.
How does the gene therapy work?
The therapy delivers working copies of the faulty gene RPE 65 into the retina, encased in a harmless virus which helps production of the essential protein for healthy vision.
How much does the Luxturna treatment cost?
The treatment costs Ā£600,000, but the NHS has a discounted price agreement with the manufacturer.
Who can benefit the most from this therapy?
Children and others with similar eye conditions may benefit the most, as it can halt sight loss before permanent damage occurs.
What changes did Jake experience after the therapy?
Jake experienced improved peripheral vision and being able to see facial features.
What impact did the therapy have on Matthew from London?
Matthew noticed improvements in independence and regained some vision enabling daily activities without assistance.
Lihat lebih banyak ringkasan video
- Luxturna
- gene therapy
- inherited eye condition
- RPE 65 gene
- tunnel vision
- NHS
- novartis
- vision improvement
- retina
- sight loss prevention