•   
  •   
  •   

Technology Could a gene therapy cure dementia?

16:55  14 january  2020
16:55  14 january  2020 Source:   qz.com

'Designer babies' could be just two years away, expert claims

  'Designer babies' could be just two years away, expert claims Genetically-modified babies are "highly desirable" to help protect people from disease and could be created ethically within two years, according to a new scientific paper. © Shutterstock Designer babies could be just two years away, a new research paper has found. Gene editing now presents such low risks that it could be used in human embryos, according to an analysis by Kevin Smith, a bioethicist at Abertay University in Scotland, published last week in the journal Bioethics. require(["medianetNativeAdOnArticle"], function (medianetNativeAdOnArticle) { medianetNativeAdOnArticle.

What type of dementia ? In alzheimer's the brain is literally losing mass. If you could restore the brain cognitive abilities would be restored, but not memory. And quite likely not personality. Your best bet would be to catch it early and use gene therapy to arrest progression of the diseaset / condition.

Gene therapy (also called human gene transfer) is a medical field which focuses on the utilization of the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease.

a man standing on a beach: A man faces the sun rising over ocean waves.© Provided by Quartz A man faces the sun rising over ocean waves.

Scientists know a lot about the hallmarks of different types of dementia. Alzheimer’s disease is characterized by buildups of amyloid and tau proteins. Vascular dementia is the result of gnarled and broken blood vessels that normally supply oxygen to the brain. Parkinson’s disease and other Lewy Body dementias are caused by misshapen alpha-synuclein proteins in the brain.

Conventional wisdom has it that each of these dementias needs its own treatment. An anti-amyloid drug probably wouldn’t work for someone who doesn’t have amyloid buildups in their brain.

Utah Gov. Gary Herbert proposes ban on conversion therapy for minors

  Utah Gov. Gary Herbert proposes ban on conversion therapy for minors Utah Gov. Gary Herbert on Tuesday proposed a rule banning conversion therapy for minors in the state. require(["medianetNativeAdOnArticle"], function (medianetNativeAdOnArticle) { medianetNativeAdOnArticle.getMedianetNativeAds(true); }); The rule comes amid ongoing discussions in the state to ban the widely discredited practice that seeks to change a person's sexual orientation or gender identity. Earlier this year, a similar bill banning the practice for minors, presented by Republican state Rep. Craig Hall, stalled in the state's legislature.

Can gene therapy cure dementia ? Gene therapy versus Immunotherapy in Alzheimer's patients? Which one would potentially bring us closer to a cure ? And the brain is a difficult target because it’s hard to disperse a gene therapy vector evenly throughout a solid tissue (as opposed to targeting

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy , including

But to this day, there are no definitive treatments—or preventive measures—for any of the dozens of dementias out there. Which has led some researchers to take a more systematic approach: What if there were a single mechanism in the brain that, when faulty, leads to all kinds of dementias? And what if this mechanism, like a switch, could be flipped off?

That’s the thinking of Michael Fossel, the founder of the Michigan-based biotech startup Telocyte, which is developing treatments for Alzheimer’s. Today (Jan. 14), Fossel published a review article (paywall) postulating that Alzheimer’s and other dementias are caused by a failing of a workhorse class of brain cells called glia. He also proposes that he and his colleagues at Telocyte, founded in 2015, have a solution: a gene therapy that could target these cells to keep dementia at bay.

Police Therapy Dog Accused Of Stealing Toys Donated For Children

  Police Therapy Dog Accused Of Stealing Toys Donated For Children A therapy dog named Ben who works for a Massachusetts police department isbeing dogged with allegations he's a thief.The Franklin Police Department has been collecting toys for kids for the SantaFoundation, a local charity."We've had several officers that have worked very hard to make sure that someof the kids in town and the community that have needs will have something toopen on Christmas," Deputy Chief James Mill told news station WFXT TV. require(["medianetNativeAdOnArticle"], function (medianetNativeAdOnArticle) { medianetNativeAdOnArticle.

Can dementia be cured ? Sign up for dementia emails. If you have a dementia diagnosis or are worried about memory problems, you can help scientists understand more about the disease, and develop future treatments, by taking part in research.

The FDA recently approved CAR-T therapy for treating certain types of cancer. Learn more by watching "NOVA Wonders: Can We Make Life?" at

The paper is theoretical—it’s a review, so it’s not presenting any original data. It’s a new way of thinking, and a bold proposition. “It’s encouraging to see individuals like Dr. Fossel pulling together research and trying to come up with new theories,” says Rebecca Edelmayer, the director of scientific engagement at the Alzheimer’s Association. The Alzheimer’s Association is a nonprofit and publisher of Alzheimer’s and Dementia, the journal in which Fossel’s review article appeared.

But while theories are important, Edelmayer says, “they also need to be tested.” Gene therapies are still relatively new. And there’s reason to wonder about the safety of the gene the therapy would introduce: one that codes for the enzyme telomerase. Before scientists can even begin to test Fossel’s systematic theory of dementia, they’ll need a lot of data demonstrating its safety.

2019: the year gene therapy came of age

  2019: the year gene therapy came of age In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome. © YinYang / IStock.com For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

Gene therapy in Parkinson's disease consists of the creation of new cells that produce a specific neurotransmitter (dopamine), protect the neural system, or the modification of genes that are related to the disease. Then these cells are transplanted to a patient with the disease.

Gene therapy . Now I'm not talking about support groups for people who can 't fit into the same pants they wore in high school. I'm talking about the Currently there are a few different ways to use gene therapy to battle cancer. One way is just to remove the mutated genetic material and replace it with

Is telomerase worth exploring for dementia?

Telomerase has been a focus of longevity research for years. It’s an enzyme that lengthens telomeres, which are the genetic caps on the end of our chromosomes. Every time cells divide, telomeres shorten—and when telomeres have been sufficiently shaved away, cells enter a state called senescence and stop dividing. Then, they self-destruct.

Shorter telomeres have been correlated with a whole host of age-related health issues: cancer, diabetes, and even forms of dementia. But it’s not the telomeres themselves that cause these issues, Fossel suggests. “As my telomeres shorten, there are a lot of other things going on, too,” he says.

The relative length of telomeres, we know, sends a signal to the rest of the cell’s DNA. As telomeres shorten with cell replication, cells change the way they carry out other genetic instructions, which can result in shoddy protein production. It’s a process called the telomere positron effect (paywall), and scientists still don’t fully understand it.

Fossel posits that when telomeres shrink in microglial cells, part of the brain’s immune system, other critical parts of their DNA degrade, too—and that genetic damage can result in many different dementias.

Gene Simmons sets Twitter ablaze with odd eating habit

  Gene Simmons sets Twitter ablaze with odd eating habit Gene Simmons does what to his cereal!The KISS rocker posted a photo to Twitter on New Year's Day of his breakfast, clearly showing ice cubes floating in the bowl of milk, adjacent to what appears to be Frosted Mini Wheats mixed with Oreo O's.

For decades we have been hearing about the promise of gene therapy – modifying someone’s DNA to correct errors that cause disease. It’s caused by mutations in a single gene that contains the instructions our bodies need to produce a protein called Factor VIII, which allows our blood to clot.

There’s no cure , and you can ’t change your genes , either. The clinical trial, led by Ronald Crystal at Weill Cornell Medicine in Manhattan, is a novel tactic against dementia as well as a new twist for gene therapy . But common diseases don’t have singular causes, so gene therapy has never seemed as

Telocyte’s gene therapy would aim to rebuild those glial telomeres. That would involve sending an active copy of the telomerase gene, TERT, into the cerebrospinal fluid, carried by a virus. The virus, which should be otherwise benign, isn’t great at getting genetic material into specific cells: in mouse models, about 5% of the total therapy winds up in neurons, to no lasting effect, and about 1% winds up in microglial cells, Fossel says. But even with the TERT gene just floating around in the glial cell for a few weeks or months, it might be enough for telomerase to lengthen those end caps and trick the cell into expressing genes like it did in its younger days.

Usually, gene therapies work by introducing new genetic material that replaces a person’s faulty or missing genetic code. Telocyte’s gene therapy, however, wouldn’t be replacing a gene: It’d just be giving glial cells another copy of one they already have. All of our cells have the TERT gene embedded in their chromosomes. But the vast majority of cells (save for red blood cells, sperm or egg cells, and cells along parts of the digestive tract) have the gene switched permanently off.

That’s for good reason: Telomerase is active in most forms of cancer. Which is why many scientists fear that inserting a gene that codes for telomerase—like Telocyte’s gene therapy—risks causing cancer

Gene Simmons sets Twitter ablaze with odd eating habit

  Gene Simmons sets Twitter ablaze with odd eating habit When she and her husband get trapped in the snow over the holidays, the "Crazy Ex-Girlfriend" star calls upon the internet for a miracle!

Gene therapy has been promoted as a promising technique for many different conditions. A very small trial of gene therapy for Alzheimer’s disease has shown beneficial effects - slowing the progression of the disease by about 50%. In this trial, genetically modified cells were injected directly into the brain.

Gene Therapy Breakthrough Could ' Cure ' Blindness Doctors who injected a genetically-modified virus into the eyes of blind patients discover it significantly

“My main concern is its safety,” says Jue Lin, molecular biologist at the University of California San Francisco whose work focuses on studying telomere length and stress levels over time. “We don’t know whether the over-expression of telomerase will increase the risk of cancer.” In the brain, particularly in the glial cells that Fossel’s proposed gene therapy would target, the cancer in question would likely be glioblastoma, a ravenously growing brain tumor.

Mouse models using telomerase gene therapy in the brain have been promising, with no notable incidence of cancer—but those experiments are imperfect. Mice express telomerase differently than humans do, Lin explains: They have a lot more telomerase, in more tissues than humans. Mice also don’t live as long as we do, and cancer takes a long time to develop, Lin says.

And gene therapies carry the risk of a dangerous immune reaction to the virus carrying the therapeutic gene. The viruses used in gene therapy today—and the one Fossel proposes using—should be safer than the ones used in the early days of gene therapy. Adeno-associated virus, or AAV for short, should elicit only the tiniest of immune responses. But scientists have recently voiced concerns about the long-term safety of gene therapies using AAV.

Are the risks worth it?

Given the risks, there’s disagreement over whether the telomerase approach is worth pursuing. “It’s important and interesting to have an additional hypothesis,” says Diego Forero, a researcher at the School of Health Sciences at the Fundación Universitaria del Área Andina in Colombia. His work, which is independent of Fossel’s, focuses on exposing astrocytes, a type of glial cell in the brain, to telomerase, to see how they’ll react. He’s found that telomerase is involved in other cellular functions, like a cell’s metabolism. In his opinion, it’s too early to say that Fossel’s theory should be tested.

9 Celeb Best Friends Who Were Nominated for Golden Globes the Same Year

  9 Celeb Best Friends Who Were Nominated for Golden Globes the Same Year We can't get enough of these dynamic duos — and neither can the Hollywood Foreign Press

Over the past decade, researches have been testing gene therapy on blind dogs in attempt to restore vision. Earlier this week, six patients in Oxford had

A year after gene therapy treatment, people with hemophilia A are showing normal levels of clotting factor and big reductions in bleeding. The search for a cure for the most common form of hemophilia seems to have taken a giant leap forward Could the insertion of a missing gene cure hemophilia?

Rather than focusing on the potential therapeutic application of telomerase in brain cells, Forero is interested in more basic, exploratory research. He thinks that applying it to a specific target—like a cure for dementia—wouldn’t tell scientists enough about all the ways telomerase could affect brain cells.

Those calls for prudence can be frustrating for dementia patients facing a dearth of options. Even with no immediate plans to conduct clinical trials, Fossel says he has already had some 200 people with mild to moderate dementia reach out to him as willing participants. They’re ineligible for most other clinical trials for dementia therapies, which tend to seek out participants who have risk factors of the disease but minimal symptoms—or none at all.

“People have faced terrible disease and said ‘I’m going to take my chances,’” says Arthur Caplan, a bioethicist at New York University’s Langone Medical Center. With vulnerable populations desperate for treatment, peer review from independent scientists becomes even more important. It’s critical that the data and research are conducted by parties that don’t have a vested financial interest in a certain outcome.

These studies also need to have strong institutional review boards, Caplan says. These boards are required any time researchers are conducting experiments with human subjects—especially when the risks are so high.

Libella Gene Therapeutics, a Kansas-based biotech startup, is beginning a clinical trial for a telomerase gene therapy to treat broad aging this year. However, it’s taken its work to Colombia, where the standards for institutional review boards aren’t as high as they are in the US. It’s a tactic informally known as “IRB shopping,” and it raises eyebrows in the research community.

“We’re always open to new ideas and novel ways [to treat dementia,” Edelmayer says. “We have to leave no stone unturned.” But, she continued, one of the biggest things we want to see is not just theories. We want to see them tested.”

At 16, She’s a Pioneer in the Fight to Cure Sickle Cell Disease .
BOSTON — Helen Obando, a shy slip of a girl, lay curled in a hospital bed in June waiting for a bag of stem cells from her bone marrow, modified by gene therapy, to start dripping into her chest. The hope was that the treatment would cure her of sickle cell disease, an inherited blood disorder that can cause excruciating pain, organ damage and early death.

—   Share news in the SOC. Networks

Topical videos:

usr: 1
This is interesting!