CAN YOU HEAR ME NOW?????...................
Posted on 11/07/2023 11:41:39 AM PST by Red Badger
Four children born with a rare form of deafness are now able to hear, thanks to a new gene therapy being trialed in China. In addition to helping others with the same condition, this treatment could one day lead to gene therapies for other, more common causes of deafness.
The challenge: Every year, about 6,000 babies are born in the US alone with some level of permanent hearing loss. The problem, in rare instances, lies in mutations in the gene that codes for the otoferlin (OTOF) protein, which helps hair cells in the inner ear transmit sounds to the brain for processing.
Cochlear implants — devices that send sound-simulating signals to the brain — can often help children with mutated OTOF genes cope with their hearing loss, but they can’t actually reverse deafness or replicate the experience of natural hearing.
What’s new? A new gene therapy designed to reverse hearing loss caused by faulty OTOF genes has now been tested in five children in China — and four of them are now able to hear.
“Before the treatment, if you put them in a movie theater with the loudest sound, they wouldn’t hear it,” Zheng-Yi Chen, an associate professor at Harvard Medical School, who helped design the study, told MIT Technology Review. “Now they can hear close to normal speech, and one can hear a whisper.”
The children who responded to the treatment have about 60-65% of normal hearing, according to lead researcher Yilai Shu, a professor in the department of otolaryngology at Fudan University in Shanghai.
How it works: The gene therapy uses a harmless virus to deliver unmutated copies of the OTOF gene into the inner ear.
Because this gene is too large to fit into the virus particle, the scientists split it into two parts. They filled millions of copies of the virus with the partial genes and then injected them directly into the cochlea.
Once in the ear, the two parts of the gene joined together and began instructing ear cells to produce the OTOF protein. It’s not yet known how long the effect will last, but it’s possible a single treatment could be enough to cure the problem permanently.
More to come: Shu presented the cases of the first five children to receive the gene therapy at a meeting of the European Society for Gene and Cell Therapy in Brussels on October 27. His team has yet to publish their work, though, so details are scarce.
That means we don’t know for sure why one child didn’t respond to the treatment, but MIT Tech Review reports that they may have had preexisting immunity to the carrier virus. If that’s the case, packaging the gene therapy in a different virus might be enough to reverse hearing loss for that child and others like them.
The other guys: Shu’s team isn’t the only one attempting to reverse hearing loss by targeting OTOF mutations.
Regeneron Pharmaceuticals is also developing a gene therapy that uses a virus to deliver healthy copies of the OTOF gene into the inner ear. On October 26, it announced that the first child to receive the therapy has experienced improvements in hearing (though it didn’t say by how much). That trial is ongoing.
Eli Lilly subsidiary Akouos is developing a gene therapy for OTOF-caused deafness, too. It hasn’t tested the therapy in people yet, but is currently recruiting participants for a clinical trial in the US.
The big picture: As for why multiple teams of researchers are focusing on this relatively rare mutation, rather than a more common cause of hearing loss, Manohar Bance, chief investigator for the Regeneron trial, said that it’s essentially the low-hanging fruit of genetic hearing loss.
“Gene therapy for otoferlin deficiency is the right starting point for young children because it’s among — if not the most — simple approaches for treating hearing loss,” said Bance, explaining that everything else in the child’s ear should be functioning normally.
“It’s really important that we get the first gene therapy treatment right because it will allow us to proceed to treating other genetic conditions,” he added.
CAN YOU HEAR ME NOW?????...................
Eh?
*
CRISPR kids!
Curing deafness, blindness, handicaps? There are activists against that.
GENE THERAPY: A THREAT TO THE DEAF COMMUNITY?
https://impactethics.ca/2017/03/02/gene-therapy-a-threat-to-the-deaf-community/
Will curing the deaf lead to ‘cultural genocide’?
https://bioedge.org/enhancement/will-curing-the-deaf-lead-to-cultural-genocide/
Why Deaf People Oppose Using Gene Editing to “Cure” Deafness
https://www.discovermagazine.com/health/why-deaf-people-oppose-using-gene-editing-to-cure-deafness
The great thing about these medical experiments on Chinese children is that it gives these kids a break from their regular jobs of making I-Phones. It must get monotonous doing that repetitive assembly work on a daily basis.
Great for kids, maybe.
Not much use for us geezers with loss from damaging noises?
It possibly could be..................
Thanks for the information , RB.
Being profoundly hearing disabled I find this interesting. Rush Limbaugh said it best. “A hearing disability is one where people get mad at YOU.” So true.
“Because this gene is too large to fit into the virus particle, the scientists split it into two parts.”
So real scientists (not federally funded ‘yes’ men) can divide a gene into parts, piggy-back the parts on a virus, inject the whole mess into the body where something tells each virus ‘A’ to find a virus ‘B’ and assemble the gene parts back together.
To me that is truly mind-blowing.
....and they are Chinese.................
Do these genes make me look fat?......................
I only wonder if fetal stem cells are involved.
Not being a doctor, I have asked if gene therapy might be used to cure people. Gotta replace the defective genes and that could be done using modified viruses or something like it.
Gotta replace all of the defective genes in the body, I guess. So that would take a long time.
But maybe we could increase the life expectancy of humans that way. “And if we could do it, should we?”
And don't allow it to escape because no telling what it might do in a different human.
The real deal will come when they can regrow the cilia in the inner ear of older people with hearing loss.
I’ve actually seen phase two clinical trials for procedure so its not too many years off
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