IT SOUNDS like science fiction, and for years it seemed as though it was just that: fiction.
But the idea of gene therapy—introducing copies of healthy genes into people who lack them, to treat disease—is at last looking as if it may become science fact.
The field got off to a bad start, with the widely reported death of an American liver patient in 1999. In 2003 some French children who were being treated with it for an immune-system problem called SCID developed leukaemia. Since then, though, things have improved. Indeed one procedure, for lipoprotein lipase deficiency (which causes high levels of blood fats, with all the problems those can bring), has been approved, in Europe, for clinical use.
The most recent success, announced last month in the Lancet, was of an experimental treatment for choroideremia, a type of blindness. This is caused by mutation of the gene for a protein called REP1. Without REP1, the eye’s light receptors degenerate. Robert MacLaren of Oxford University used a virus to deliver working versions of the REP1 gene to the most light-sensitive part of the retina. Five of the six participants in the trial duly experienced an improvement in their sensitivity to light. Two were so improved that they could read more letters than previously on a standard eye chart.
Dr MacLaren’s work complements that of Albert Maguire and Jean Bennett at the University of Pennsylvania, who use gene therapy to treat another eye disease, Leber’s congenital amaurosis. A defective version of a gene called RPE65 means that, in this condition, retinal cells are starved of vitamin A, which also causes blindness. Putting normal copies of RPE65 into the retina leads, as with REP1, to greater light sensitivity and—sometimes—clearer vision.
The Latest on: Gene therapy
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The Latest on: Gene therapy
- More children gain hearing as gene therapy for profound deafness advanceson May 10, 2024 at 3:08 pm
Enlarge / Opal Sandy (center), who was born completely deaf because of a rare genetic condition, can now hear unaided for the first time after receiving gene therapy at 11-months-old. She is shown ...
- Gene Therapy Trial Restores Hearing in Baby Girlon May 10, 2024 at 10:30 am
A gene therapy trial at Cambridge University Hospitals in the UK has improved the hearing of an 18-month-old baby girl, born deaf due to auditory neuropathy.
- Toddler born deaf can hear after gene therapy trial breakthrough her parents call "mind-blowing"on May 10, 2024 at 8:26 am
The parents of a U.K. toddler say it's "absolutely mind-blowing" to see their daughter, enrolled in a gene therapy trial, hear for the first time.
- Gene therapy brings hearing to baby girl born deafon May 10, 2024 at 7:50 am
Opal Sandy was born into a world she could not hear. The British baby girl, now 18 months old, had a rare genetic condition called auditory neuropathy that interrupted nerve impulses that travel from ...
- Child who was born deaf can now hear whispers and parents clapping for first time thanks to ‘mind-blowing’ gene therapyon May 10, 2024 at 7:36 am
A deaf child has received a new gene therapy treatment with “mind-blowing” results that have allowed her to hear for the first time. Opal Sandy, from Oxfordshire, UK, is the first British patient and ...
- Baby Girl Born Deaf Gains Hearing After Gene Therapyon May 10, 2024 at 6:09 am
The gene therapy Opal received involved the transmission into her right ear of a working copy of the OTOF gene, delivered during surgery into the ear's cochlea via a harmless virus called AAV1. The ...
- British baby girl becomes world’s first to regain hearing with gene therapyon May 10, 2024 at 5:57 am
This therapy enabled the baby, who was born with a rare genetic condition that impaired ear function completely, to hear even faint sounds.
- Deaf Baby Girl Hears for First Time in 'Mind-Blowing' Gene Therapy Trialon May 9, 2024 at 11:47 am
Hearing loss affects about 1.5 billion people, while congenital deafness—hearing loss present at birth—affects approximately 1.7 out of every 1,000 children born in the U.S. The OTOF-related deafness ...
- Gene therapy breakthrough allows toddler born deaf to hearon May 9, 2024 at 10:03 am
A British toddler born with a genetic condition resulting in deafness can now hear in one ear thanks to a pioneering new gene therapy treatment.
- Deaf Toddler Hears For The First Time After Pioneering Gene Therapyon May 9, 2024 at 9:34 am
A deaf toddler can now hear normally in one ear after being treated with a pioneering experimental gene therapy in a clinical trial.
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