gene therapy

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Gene Therapy Offers 'Astounding' Results in Deaf Kids

Small group of children in China see marked improvement in hearing after a gene mutation fix

(Newser) - A handful of children in China are experiencing sound for the first time, thanks to scientists who employed gene therapy to help alleviate their hereditary hearing issues. The Guardian reports on the "astounding" research, published Wednesday in Nature Medicine , in which doctors at Shanghai's Fudan University treated five...

She Was Born Deaf. Then a 'Spectacular' Turnaround

After gene therapy, Opal Sandy of the UK hears well, and loves making a racket

(Newser) - Opal Sandy was born deaf as a result of auditory neuropathy, a condition that disrupts the way sound is conveyed to the brain from the inner ear. But at 18 months, the UK toddler can now "hear almost perfectly after groundbreaking surgery that took just 16 minutes," the...

It Feels 'Like Being Stabbed All Over.' A New Cure Is Closer

Panel clears CRISPR gene-editing product for sickle cell disease patients; FDA to weigh in

(Newser) - Sickle cell disease is a painful, debilitating, and possibly deadly inherited disorder that currently can only be remedied with a bone marrow transplant. Now, Boston's Vertex Pharmaceuticals hopes exa-cel, its treatment developed with CRISPR Therapeutics, will soon be approved by the FDA following a thumbs-up from a panel of...

Man Behind Gene-Edited Babies Is Back

He Jiankui aims to study gene therapy for Duchenne muscular dystrophy in Hong Kong

(Newser) - A Chinese scientist who set off an ethical debate five years ago with claims that he made the world’s first genetically edited babies said Tuesday that he hopes to research rare hereditary diseases in Hong Kong, per the AP . He Jiankui shocked the world in 2018 when he announced...

The FDA Has Just Approved a Drug That Costs $3.5M a Dose

Hemgenix is a one-time treatment for the blood-clotting disorder hemophilia

(Newser) - US health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. The FDA cleared Hemgenix: It's an IV treatment for adults with hemophilia B, the less common form of the genetic disorder, reports the AP . Currently, patients receive...

Doctors Say Gene Therapy Cured 2 of Cancer

CAR-T cells attacked cancer and stayed in the body of men with leukemia

(Newser) - In 2010, doctors treated Doug Olson's leukemia with an experimental gene therapy that transformed some of his blood cells into cancer killers. More than a decade later, there's no sign of cancer in his body. The treatment cured Olson and a second patient, reported University of Pennsylvania doctors,...

Reversed Aging Is Close, Geneticist Says

Process works on mice and has moved on to a spaniel

(Newser) - So far, aging has been reversed only in mice. We'll know soon if the process works for spaniels. In less than 20 years, George Church figures, it could be available to humans. The geneticist works with 100 or so scientists in a Harvard Medical School lab to make that...

New Drug Could Cure Horrible Disease. Now, Brace Yourself
New Drug Could Cure Horrible
Disease. Now, Brace Yourself
in case you missed it

New Drug Could Cure Horrible Disease. Now, Brace Yourself

Novartis AG is expected to sell Zolgensma for $2M per treatment

(Newser) - A new wonder drug is coming for babies with a crippling, often fatal disease. Expected price tag: $2 million per treatment, the Wall Street Journal reports. Novartis AG is expected to begin selling Zolgensma, a possible cure for spinal muscular atrophy, after the FDA approves it this month. Novartis executives...

Scientists May Have Altered DNA in Adults to Fight Disease

Researchers caution experiments are encouraging but not conclusive

(Newser) - Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease, though it's too soon to know if this will help. Preliminary results suggest that two men with a rare disorder now have a corrective gene at very...

Company Says Its Treatment for Blindness Will Cost $850K

Spark Therapeutics reveals Luxturna gene therapy will cost $425K per eye

(Newser) - It's a gradual and cruel fate: an inherited form of vision loss that typically ends in blindness and has had no cure. But for those with mutations in a gene known as RPE65 comes a balm. The FDA in December approved Luxturna, a new gene therapy that can treat...

Scientists 'Toy With Mother Nature' in 'Bold' Genetic First

For first time, scientists attempted gene editing inside a live human to try to cure genetic disease

(Newser) - Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to cure a disease. The experiment was done Monday in California on 44-year-old Brian Madeux, the AP reports. Through an IV, Madeux, who has a metabolic...

Doctors Find Way to Halt Deadly Child Brain Disease

Gene therapy is 'curative' for ALD, says doctor

(Newser) - Without a risky bone-marrow transplant before symptoms appear, children with brain disease ALD can expect to live no longer than five years as nerve cells in the brain die off and erase one's ability to walk, talk, and think. Even a successful transplant can result in permanent disabilities, reports...

New Cancer Treatment Called 'Most Exciting Thing I've Seen'

FDA unanimously approved it

(Newser) - "This is the most exciting thing I've seen in my lifetime," Dr. Timothy Cripe said Wednesday after voting in favor of a groundbreaking new cancer treatment. The oncologist and nine other members of a Food and Drug Administration panel voted unanimously to recommend approval of the treatment,...

US Panel: OK to Make 3-Parent Embryos —Male Ones

But there are still legal roadblocks to controversial process

(Newser) - A US National Academy of Medicine committee said Wednesday the FDA should approve clinical trials for three-parent fertilization in human embryos—with some caveats, Nature reports. In its report , the committee recommended such trials be limited to women at risk of passing along "serious mitochondrial disease" to their kids,...

Gene Injection Gives Pigs 'Biological Pacemakers'

Heart cells were reprogrammed to beat normally

(Newser) - A promising experiment on pigs could help save the bacon of people with heart trouble who are having problems with electronic pacemakers. Researchers at the Cedars-Sinai Heart Institute injected genes into the hearts of pigs with irregular heartbeats, reprogramming regular heart cells into pacemaker cells that temporarily restored a normal...

Scientists Cure Red-Green Color Blindness in Monkeys

Adult brain can re-wire to use new vision input: study

(Newser) - Scientists have cured red-green color blindness in monkeys, the Times of London reports. Researchers injected a virus containing L opsin, a gene that regulates the production of the red-sensitive light receptor—known as a “cone”—into the retina of red-green colorblind adult monkeys, according to a study published...

Cancer Treatment May Have Cured Man's AIDS

After marrow transplant, patient stays virus-free

(Newser) - A German doctor has inspired hope for a new approach to AIDS treatment with his handling of a leukemia case, the Wall Street Journal reports. Because the patient also had AIDS, Gero Hütter looked for a bone marrow donor with a specific mutation that seems to stymie the HIV...

Genetic Hiccup Causes Obesity In Mice: Study

Evolutionary relic in humans could be fixed by drug or gene therapy

(Newser) - Scientists have discovered an immune system pathway in the brain that they think is the root cause of diseases related to obesity, Reuters reports. When mice were overfed, the hypothalamus secreted a compound that suppressed the conversion of food into energy, and led to inflammation associated with heart disease and...

Gene Breakthrough Aids Blind
 Gene Breakthrough Aids Blind 

Gene Breakthrough Aids Blind

Experimental therapy improves sight

(Newser) - A rare and incurable congenital disease which causes blindness has been successfully treated for the first time using gene therapy, Reuters reports. Doctors inserted a healthy gene directly into one eye of patients suffering from Leber congenital amaurosis using a genetically engineered cold virus. Patients reported an improvement in their...

Skin Stem Cells Cure Sickle Cell in Mice

Latest advance in revolutionary research

(Newser) - In the latest advance in revolutionary new stem cell research, scientists have cured sickle-cell anemia in mice using stem cells made from skin. It's the first proof that such cells can be used to cure hereditary diseases. But because the cure involved the use of viruses to alter DNA, significant...

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