British doctors have managed to cure the blindness of children born with a rare disease thanks to a new gene therapy, reports The Guardian.
Children’s meetings suffered from congenital amurosis (ACL), a serious form of retinal dystrophy that leads to loss of vision due to a defect in gene Aip1. The people in question are legally blindly declared by birth.
But four children with this genetic disease can now see forms, find toys, recognize the face of their parents and, in some cases, even read and write. This was possible thanks to the doctors who injected healthy copies of the APL1 gene in their eyes during a surgical operation that only lasted.
“The results for these children are extremely impressive and show the power of gene therapy to change lives,” said Professor Michel Michaelides, a specialist in retinal consultants at the Moorfields Eye hospital and an ophthalmology professor at the UCL ophthalmology institute (UCL ( The University College of London).
For the teacher, it is “for the first time, an effective treatment against the most serious way of child blindness and a possible paradigm change towards treatment from the early stages of the disease.”
A treatment managed only in one eye
Four children, age of one to two years, from the United States, Turkey and Tunisia, were selected by specialists from Moorfields and UCL in 2020. The operations were carried out at the Great Ormond Street hospital in London.
These healthy copies of the Aip1 gene, contained in a harmless virus, were injected into the retina, the light sensitive tissue layer located on the back of the eye. To avoid any serious risk, treatment has only been administered only in an eye per patient. Then, the children were followed for five years before the results were published in Lancet magazine.
Professor James Bainbridge, retinal surgery consultant at Moorfields and a professor of retina studies at the UCL Ophthalmology Institute, said that children born with ACL can only distinguish light and darkness, and that the little girl loses in a few years vision they have.
“Some children (who have followed the editor’s note) can even read and write after the intervention, which is not expected in this situation, without treatment,” he develops.
Other seven children currently treated
The parents of one of the children, Jace, talk about “quite incredible” results and said they were lucky to have benefited from it. His son, who was two years old at the beginning of his treatment and now has six, can now collect small objects on the floor and identify toys from distance.
“I remember that I cried and I had become very emotional,” said his father Brendan, “from there, he became quite incredible.”
Since the four children received therapy, another seven have been treated at Evelina Children’s Hospital in London by specialists from the St Thomas Hospital, Great Ormond Street and Moorfields.
Source: BFM TV
