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Posted on 05/02/2026 6:36:36 AM PDT by Twotone
Deaf children can now hear thanks to a new treatment that repairs a defective gene. Researchers associated with the biotech company Regeneron Pharmaceuticals unveiled the successful gene therapy last week. One patient, who was treated at 18 months old, Travis Smith, can now hear. Overall, 80 percent of participants (aged 10 months to 16), saw significant improvement in hearing, and 42 percent achieved normal hearing, including the ability to hear whispers. The Food and Drug Administration (FDA) has already approved the treatment.
Medical research on ameliorating physical and intellectual maladies continues apace. For example, researchers at Harvard Medical School reported earlier this month that they have made progress in silencing the extra chromosome that causes Down syndrome using the genome editing technique CRISPR.
Down syndrome is a genetic condition in which a person has an extra copy of chromosome 21. While its severity varies, people with the condition experience lifelong intellectual disability and developmental delays, along with heart and digestive system problems and a significantly higher risk of Alzheimer's disease.
CRISPR techniques have recently been successfully applied to treating other genetic conditions, including sickle cell anemia and a rare inborn error of metabolism of the urea cycle, which causes life-threatening accumulation of ammonia in the bloodstream.
Keeping in mind that the new Harvard study is a very preliminary cell-based experiment, the researchers believe their work "paves a road for therapeutic treatment for DS [Down syndrome]." It's likely to be a long road, but the development of a way to successfully correct this genetic error raises some interesting ethical issues.
Currently, about 67 percent of women in the United States who receive a positive prenatal test for Down syndrome choose to terminate the pregnancy. These decisions might have been changed if the extra chromosome could be safely silenced in fetuses before birth.
But what about offering the CRISPR treatment to children and adults with Down syndrome?
University of Warwick bioethicist Felicity Boardman has suggested that the development of genetic treatments for various conditions, such as deafness, dwarfism, and blindness, would "convey and perpetuate negative views about the particular disabling conditions they are targeted towards, and, by extension, people who currently live with those conditions." The fact of disability does not diminish the inherent moral worth of any individual. And thanks in large part to their activism, many barriers that once prevented disabled folks from more fully participating in society have been lowered and ameliorated.
As the success of the Regeneron treatment for deafness, however, shows, many people with specific disabilities (and their guardians) already seek out corrective treatments when they become available. For example, some 315,000 deaf Americans have chosen to use cochlear implants to enable them to hear. Genetic treatments for blindness are also being developed. Adult deaf and blind people can give their informed consent to the corrective measures. Of course, in the case of the deaf infants and minor children treated using the Regeneron gene therapy, their parents did.
Already treatments are recommended to mend infirmities that often accompany Down syndrome, such as surgery to repair heart defects. Silencing the extra chromosome in children and adults to treat heart and digestive maladies, or even Alzheimer's disease risks associated with Down syndrome, does not seem especially problematic ethically.
But who can ethically make decisions that affect intellectual disabilities? After all, the effects of safely delivering effective chromosome silencing to the brains of a child or an adult with Down syndrome would be even more profound than restoring hearing or sight. People with Down syndrome, whose extra chromosome was silenced in their brain cells, might undergo a change in cognition, something like what Charlie Gordon experiences in the sci-fi story Flowers for Algernon, but in this case, the change would be permanent.
There are now reams of studies and reports on how researchers and clinicians should go about obtaining informed consent from people with intellectual disabilities. Most often, the ultimate decision to treat or not rests with the parent or legal guardian of the patient. Various surveys find that some parents of children with Down syndrome would be interested in genetic interventions that improve their child's cognitive ability. In Flowers for Algernon, Gordon's estranged sister provided the consent for the experimental treatment that (albeit temporarily) boosts his IQ from 68 to over 185.
Since Down syndrome is implicated in their very identity, successfully treated folks would in some very real sense no longer be the persons that they were. Perhaps one fruitful way to think about the ethical implications of using chromosome silencing is to ask yourself if you would accept a safe treatment that would significantly boost your IQ or improve your memory? (I, for one, would happily accept a cure for my aphantasia.)
In Flowers for Algernon, Gordon writes in his initial entry of his progress report diary, "I hope they use me. Miss Kinnian says maybe they can make me smart. I want to be smart." Becoming smarter did not ultimately make Gordon happier; he did not have time to adjust to his changed mental and emotional states. Assuming chromosome silencing improves cognition, treated Down syndrome folks, unlike Gordon, would have that time. And research suggests that higher intelligence correlates with greater happiness.
"Should we be using CRISPR for diseases or syndromes that are compatible with life, like Down syndrome?" asked Harvard physician Neal Baer at a symposium last year. "Who is going to make those decisions?" The ethical answer is yes, and we should trust parents— and no one else—to make those decisions.
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“Who is going to make those decisions?” The ethical answer is yes, and we should trust parents— and no one else—to make those decisions.
The same parents whom 67% of now choose to abort based upon prenatal diagnosis of Down’s?
University of Warwick bioethicist Felicity Boardman has suggested that the development of genetic treatments for various conditions, such as deafness, dwarfism, and blindness, would "convey and perpetuate negative views about the particular disabling conditions they are targeted towards, and, by extension, people who currently live with those conditions." The fact of disability does not diminish the inherent moral worth of any individual.We anti-abortion people of course do not claim that disability diminishes a person's inherent moral worth. Quite the contrary! Indeed, that truth is in perfect alignment with our moral position - that abortion (whatever the motivation) is evil!
However, the quite independent argument that the development of therapies and/or treatments which alleviate or even reverse disabilities might somehow "convey and/or perpetuate" negative stereotypes about disabled people is specious and abhorrent!
Regards,
If genetic/chromosome treatments can also eliminate homosexuality, would the gay community and liberals object to the treatments, and even prevent them from being developed?
CRISPR actually can perform gene therapy. Genetic strings can be cut, defective DNA code removed, and new sequences insterted using it.
Contrast that with the hysteria over mRNA technology which can’t alter DNA at all, despite the constant BS from Qtard quacks.
If genetic/chromosome treatments could eliminate genetic heart conditions, would Conservatives object to it?
CRSPR “technology” ....not really our invention (actually, it was first discovered in bacteria). Underscores the staggering complexity of our genetic biome.
We know that on a molecular scale, as one example, there are biochemical “mistakes” made during DNA replication and sometimes the wrong nucleotide is inserted in the replicating DNA chain. Usually those mistakes are “corrected” by aptly named correction enzymes. I did mention that our genetic machinery is staggeringly complex.
The presence of biochemical replication errors may also allow the CRSPR technology to have unintended mistakes associated with it.
We need to make sure that the CRSPR technology does NOT edit other unrelated, but chemically similar, parts of the chromosome while doing its intended therapeutic function. Could be disastrous, especially if germ cell DNA is altered.
We embraced chemical “therapeutic” agents long time ago, and we know a lot about their unintended consequences. We need to be aware of the unintended genetic consequences for gene editing also.
IMHO
“convey and perpetuate negative views about the particular disabling conditions they are targeted towards, and, by extension, people who currently live with those conditions.”
This argument is intellectually bankrupt, based on a flawed premise, like all woke ideology, and would deny people the opportunity to succeed, to reach their full potential, and to experience aspects of life that are currently closed to them. Such an attitude is the worst type of paternalism.
The opportunity to help people with CRISPR based therapy is a bright spot in the world. A game changer for the individuals and families that can be helped. News of these early successes is exhilarating.
CRISPR (short for “clustered regularly interspaced short palindromic repeats”) is a natural defense mechanism found in bacteria that helps them fight off viruses.
When a bacterium survives a viral infection, it stores a snippet of the virus’s DNA in its own genome. This stored DNA, known as a “space sequence,” allows the bacterium to recognize and destroy the virus if it attacks again. Researchers adapted this immune defense system to edit DNA. They create a small piece of RNA with a short “guide” sequence that attaches (binds) to a specific target sequence in a cell’s DNA, much like the RNA segments bacteria produce from the CRISPR array. This guide RNA also attaches to the Cas9 enzyme. When introduced into cells, the guide RNA recognizes the intended DNA sequence, and the Cas9 enzyme cuts the DNA at the targeted location, mirroring the process in bacteria.
The research aimed at using CRISPR to treat Down syndrome focuses on removing the extra chromosome rather than editing individual genes on it.
In studies targeting Down syndrome, the CRISPR-Cas9 system uses synthetic guide RNAs (gRNAs) designed by researchers to specifically target unique DNA sequences on the extra (third) copy of chromosome 21. These gRNAs guide the Cas9 enzyme to create multiple cuts in the extra chromosome, causing it to break down and be removed from the cell.
Cell Sources: The DNA being edited comes from patient-derived pluripotent stem cells or skin fibroblasts (fibroblasts), which are then treated with the CRISPR-Cas9 complex in laboratory settings.Method of Action: The guide RNA directs the Cas9 enzyme to the specific sequence, cutting it 13 times, breaking it down, and enabling the cell to function without the extra chromosome.
CRISPR technology can effectively cure sickle cell disease (SCD) by modifying a patient’s own stem cells to produce healthy fetal hemoglobin, eliminating severe pain crises. The FDA-approved therapy, Casgevy, is the first CRISPR-based treatment, enabling patients with SCD to live free of vaso-occlusive crises, making it a functional cure.
This Stanford paper discusses the many anticipated slip between cup & lip. There will is much potential but the applications need to be focused.
https://law.stanford.edu/2025/11/23/cutting-to-the-core-down-syndrome-crispr-and-the-future-of-human-diversity-part-ii/
Felicity Boardman is batshit crazy: here insane assertion regarding “moral worth” could be applied to any treatment for any medical condition ...
GMOs are all the rage now. I used to tell that people once you have the opportunity to have
G.enetically
M.odified
O.ffspring
that GMOs will become highly popular and the looks I would see on people’s faces were very humorous.
It is happening.
The obvious flaws for fixing genetic problems are too irresistible to ignore, but just wait until it expands into picking hair and eye color, height in adulthood, all sorts of things.
Designer “Gucci children” will be the ultimate handbag accessory.
Devil’s avocate thinks the Pro-Life movement could embrace this.
If you could prevent an abortion by CRISPRing a down’s baby to becoming a “normal baby” and thus abortion would be avoided, why wouldn’t that be the best choice?
If you could just save one life isn’t it worth it??? Isn’t CRISPR worth it?
/devil’s advocate
You think I’m kidding. It is coming.
even worse, CRISPR could easily be abused to deliberately create monsters, sub-human drudges à la the Epsilons in “Brave New Worlds”, punish people with cruel defects, and worse ...
I see the reluctance to allow maladies to be cured to be the same as would happen if a cure for cancer was found. The people who make money on “cancer research” would come out fighting mightily to stop it. To exaggerate any possible side effects in order to stop it.
There’s too much money to be made from the disorders existence to simply allow it to be cured!
CRSPR precisely edits specific bits of genetic code in a specific gene. For genetic diseases caused by one bad gene, numerous though individually rare, they may offer fixes. At least if you can get the fix to where it’s needed soon enough. In this deafness case only the inner ear needs it. Which can be physically targeted and which minimizes the risk of that fix breaking something else. When a whole body in vivo code fix is needed fixing enough is harder and changing too much is a big risk. In some cases using CRSPR in vitro allows synthesis of a corrected gene product, which delivered like a drug may correct the problem. Sometimes the fix may need to be done before irreversible damage is done. In some cases may need gene correction pre-birth as part of an IVF process. The collective potential is great, but will need time to develop with a one size fit all solution unlikely. However I don’t see how CRSPR could help Downs. The genes are all fine qualitatively in that. The problem is quantitative, too many copies of hundreds or thousands of genes , thus making the wrong amounts of loads of stuff. No one spot of bad code to correct; you need to shut down the good code throughout the extra chromosome. And I don’t think CRSPR can tell one chromosome copy from another.
Amazing!
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