Cientistas obtêm sucesso promissor com terapia gênica para surdez na China

O Gato Laranja

Um novo estudo publicado na revista Nature mostra que uma terapia genética foi capaz de restaurar a audição em crianças e adultos com surdez congênita.  Uma única injeção melhorou a audição de todos os dez pacientes em um ensaio clínico, sem efeitos colaterais graves.  A descoberta é um passo importante para a cura da surdez genética.

Transcript

00:00Scientists achieve promising success with gene therapy for deafness in China.

00:05New hope emerges for people with congenital deafness.

00:09A groundbreaking study, published July 2 in the journal Nature, reveals that a new

00:14Gene therapy can restore hearing in children and adults born with hearing loss

00:20severe hearing loss.

00:21The research, which involved the Karolinska Institutet in Sweden and hospitals in China, showed improvements

00:27in the hearing of all 10 patients, with no significant adverse reactions.

00:32This is a major step forward in the genetic treatment of deafness, which has the potential to

00:37change the lives of children and adults, said Mauli Duan, one of the study's authors and a professor

00:42from the Karolinska Institutet.

00:45The study included 10 Chinese patients aged 1 to 24 years, all with a form

00:51of deafness caused by mutations in the Otov gene.

00:53This genetic defect prevents the production of the protein otoferlin, which is essential for transmission

00:59of sound signals from the ear to the brain.

01:02Gene therapy involves using an adeno-associated virus as a vector to deliver a

01:07functional version of the Otov gene to the inner ear of patients.

01:11The procedure is done with a single injection through the round window, a membrane at the base

01:16of the cochlea.

01:18The effect of the therapy was remarkably rapid, with most patients recovering some

01:23hearing in just one month.

01:25A six-month follow-up showed considerable improvement in all participants.

01:30With the average volume of sound they could hear dropping from 106 decibels to 52 decibels,

01:36a dramatic improvement.

01:38The treatment was most effective in younger participants, especially those between 5 and 8 years old.

01:45One of the most impressive cases was that of a 7-year-old girl who recovered almost all of her

01:50hearing in a few months and was already able to have daily conversations with his mother.

01:54However, the method has also proven effective in adults.

01:58Hearing improved greatly in many of the participants, which can have a profound effect on their quality

02:03of life, said Dr. Duan.

02:05We will now be following these patients to see how long the effect lasts.

02:10Researchers also confirmed that the treatment is safe.

02:13The most common adverse reaction was a slight reduction in a type of white blood cell,

02:18and no serious adverse reactions were reported during the 6 to 12 months of follow-up.

02:24While the Otof gene treatment is a success, researchers are already looking to the future.

02:30They plan to expand the work to treat mutations in other, more common genes that cause deafness,

02:36such as GJB2 and TMC1.

02:38Otof is just the beginning, said Dr. Duan, who is confident that in the future,

02:44patients with different types of genetic deafness will be able to receive effective treatments.

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