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11-Year-Old Boy Hears For First Time Ever Thanks To Gene Therapy Breakthrough...The procedure is now being trialed on more children.
IFL Science ^ | 24 January 2024 | LAURA SIMMONS

Posted on 01/27/2024 1:15:43 PM PST by Red Badger

The treatment introduces functioning copies of the OTOF gene, which is mutated in some people with childhood hearing loss.

Image credit: Dragon Claws/Shutterstock.com

An 11-year-old boy who was born with congenital hearing loss has become the first patient to receive a new gene therapy procedure, and it’s allowed him to hear sounds for the first time in his life.

“Gene therapy for hearing loss is something that we physicians and scientists in the world of hearing loss have been working toward for over 20 years, and it is finally here,” said Dr John A. Germiller, Director of Clinical Research in the Division of Otolaryngology at Children’s Hospital of Philadelphia (CHOP), in a statement.

“While the gene therapy we performed in our patient was to correct an abnormality in one, very rare gene, these studies may open the door for future use for some of the over 150 other genes that cause childhood hearing loss.”

The patient, Moroccan-born Aissam Dam, has an extremely rare form of deafness that is thought to affect only about 200,000 people worldwide. It is caused by a recessive mutation in the otoferlin (OTOF) gene. Dam was born with profound hearing loss in both ears, but for safety reasons, the researchers had to start by treating only one ear.

During surgery, the patient’s eardrum was partially lifted to open a tiny access window into the cochlea. A single dose of the experimental gene therapy was then inserted directly into the inner ear – a harmless viral vector containing copies of the normal OTOF gene. The idea is that having the functioning gene in place will allow the sensory cells to transmit signals along the auditory nerve to the brain, as happens in hearing people, although the scientists weren't certain it would work in someone who had been deaf for 11 years.

However, four months later, the hearing in the treated ear has recovered to only mild/moderate hearing loss, meaning that Dam can hear sounds for the first time in his life. In an interview with the New York Times, with the help of a translator, his father explained how he could hear traffic noises only days after the surgery.

“There’s no sound I don’t like,” Dam expressed through interpreters. “They’re all good.”

The surgical technique that makes this treatment possible was developed 10 years ago by Dr Germiller, and it is used for a different diagnostic procedure in young children. Combining this with cutting-edge gene therapy research from Akouos, Inc., a subsidiary of pharma giant Eli Lilly and Company, has led to the current clinical trial.

“As more patients at different ages are treated with this gene therapy, researchers will learn more about the degree to which hearing is improved and whether that level of hearing can be sustained over many years,” Dr Germiller said. “What we have learned from following this patient’s progress will help direct our efforts toward helping as many patients as we can.”

The New York Times reports that another child was similarly treated in Taiwan towards the end of last year, and the CHOP team has two further candidates lined up for surgery. A 3-year-old boy from Miami and a 3-year-old girl from San Francisco will both be treated in one ear, while their other ears have already been fitted with cochlear implants.

If the trials continue to produce such positive results, attention is likely to turn to the other 150-plus genes that are implicated in congenital hearing loss. However, the idea of providing a “treatment” for deafness at all is a controversial one, with some members of the Deaf community rejecting the characterization of hearing loss as a disability.

In cases where parents do choose to seek treatment for their children born with hearing loss, the results of this trial represent an exciting possibility for the future.

“For decades people have been saying, ‘When is this going to work?’,” Dr Margaret Kenna, an otolaryngologist and professor of otolaryngology at Harvard Medical School, told the New York Times. “I didn’t think gene therapy would begin in my practice lifetime. But here it is.”


TOPICS: Health/Medicine; History; Military/Veterans; Society
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1 posted on 01/27/2024 1:15:43 PM PST by Red Badger
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To: joe fonebone; SamiGirl; gitmogrunt; Freee-dame; ROCKLOBSTER; ryderann; Red_Devil 232; ...

Ping!................


2 posted on 01/27/2024 1:17:22 PM PST by Red Badger (Homeless veterans camp in the streets while illegal aliens are put up in hotels.....................)
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To: Red Badger

So, a proper use of Fauci’s satanic abuses?

I had an inkling that those nano-lipids and mRNA work had POSITIVE uses.

Notice what the world was exposed to, first.


3 posted on 01/27/2024 1:22:38 PM PST by Empire_of_Liberty
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To: Red Badger

Damage control.


4 posted on 01/27/2024 1:35:22 PM PST by E. Pluribus Unum (The worst thing about censorship is █████ ██ ████ ████████ █ ███████ ████. FJB.)
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To: E. Pluribus Unum

If it works on children who have never been able to hear, then it should work on adults who have lost their hearing.........


5 posted on 01/27/2024 1:57:01 PM PST by Red Badger (Homeless veterans camp in the streets while illegal aliens are put up in hotels.....................)
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To: Red Badger

It sounds like a genetic fix, which is probably a gross oversimplification.

Adults losing their hearing is probably physical damage to the mechanism of the ears.

The included image is cute, but I doubt that the cells of an afflicted person get their DNA repaired and start producing healthy cells. I won’t be surprised at all if the hearing improvement isn’t lost over time, sadly.


6 posted on 01/27/2024 2:17:22 PM PST by Empire_of_Liberty
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To: Red Badger

... procedure is now being trialed ...?


7 posted on 01/27/2024 2:38:38 PM PST by MV=PY (The Magic Question: Who's paying for it?)
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To: Red Badger
“There’s no sound I don’t like,” Dam expressed through interpreters. “They’re all good.”

Apparently he hasn't heard any country and western music yet.

8 posted on 01/27/2024 2:49:11 PM PST by AlaskaErik (There are three kinds of rats: Rats, Damned Rats, and DemocRats.)
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To: Red Badger

The videos of people, of all ages. getting cochlear implants and can now hear will make you cry with joy as you watch them absolutely overcome with happiness. It’s a total sobfest, in a good way.


9 posted on 01/27/2024 3:00:59 PM PST by Lizavetta
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To: AlaskaErik

There isn’t much ‘country’ or ‘western’ in ‘country and western’ music nowadays...........


10 posted on 01/27/2024 3:03:59 PM PST by Red Badger (Homeless veterans camp in the streets while illegal aliens are put up in hotels.....................)
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To: Empire_of_Liberty

My dentist told me yesterday that in twenty years they will simply pull rotten or broken teeth and grow new ones with stem cells. He said they’ve finished the mouse trials and are starting pig and monkey trials. Then it will be human trials. He wxpects it will be cheaper than implants.


11 posted on 01/27/2024 3:11:47 PM PST by Valpal1 (Not even the police are safe from the police!!!)
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To: Valpal1

I could absolutely go for that, assuming they’re my stem cells, or whatever.

I seem to be blessed with rotten teeth.

Actually, now that I re-read your post, I may not have any teeth in 20 years, so it may not matter.


12 posted on 01/27/2024 3:40:40 PM PST by Empire_of_Liberty
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To: Empire_of_Liberty

“Adults losing their hearing is probably physical damage to the mechanism of the ears.”

Mine happened 2 days after my vaccine because of severe ringing in my ears.

I’ve met 2 other people who this happened to post jab but VAERS reports this side effect occurred in less than 20,000 people. Yeah Right.

On the same day I also developed (I think) superior canal dehiscence syndrome. For about the first hour or two every morning when I move my eyes to the right I hear a deep clicking noise. Weird but no big deal.


13 posted on 01/27/2024 4:30:42 PM PST by lizma2
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To: lizma2

I have the clicking noise as well. I started gettin really impaired after it first started,
then the ringing, at least fifteen years ago. Now that it’s back, my hearing is worse
i need go back for new hearing aids,, Got mine at costco... perhaps I do need an Audiologist11


14 posted on 01/27/2024 5:49:00 PM PST by acapesket (all happy now?)
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