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Gene Therapy Could Be Big Advance Against Hemophilia

Gene therapy shows promise in reducing, and even halting, potentially life-threatening bleeding events in people with hemophilia, researchers report.

Hemophilia A is the most common inherited bleeding disorder, affecting one in 5,000 males worldwide. It's caused by a missing coagulation factor called FVIII.

The current standard of care involves regular infusions of the FVIII protein...

Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in Kids

THURSDAY, Nov. 18, 2021 (HealthDay News) -- Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.

In a study of eight children with the condition, called Hurler syndrome, researchers found that the gene therapy was safe over two years. It also showed potential for beating...

Mouse Study Offers Hope for Gene Therapy Against Parkinson's Disease

An experimental gene therapy to boost the effectiveness of the Parkinson's drug levodopa yielded promising results in mice, researchers report.

As the loss of dopamine-releasing neurons advances in late-stage Parkinson's, levodopa is less able to ease movement problems caused by the disease, which is a progressive disorder of the nervous system.

But a Northwestern University team fo...

Ten Years On, Gene Therapy Still Beating Most Cases of 'Bubble Boy' Immune Disease

Nine of 10 patients with so-called "bubble boy" immune disease who received gene therapy about a decade ago are still disease-free, researchers report.

The gene therapy was developed at the University of California, Los Angeles (UCLA), to treat the rare and deadly immune system disorder formally known as adenosine deaminase--deficient severe combined immunodeficiency (ADA-SCID).

It'...

Even When Undergoing Treatment, People With MS Gain From COVID Vaccines

Multiple sclerosis (MS) patients undergoing a treatment that depletes a type of immune cell that fuels MS attacks still have a strong response to mRNA COVID-19 vaccines, a new study finds.

"The message from this study is clear — it is worthwhile for patients with MS receiving [anti-CD20] treatment to get a COVID-19 vaccine, which will prevent severe illness," said researcher E. John Whe...

Pig Study Could Lead to Gene Therapy to Prevent Heart Failure

A gene therapy aimed at freeing the heart's capacity for self-repair has shown early promise in an animal study.

The study -- done in pigs -- found that the treatment approach was not only feasible, but also improved the animals' heart function after they sustained heart attack damage.

There is a long way to go before a similar gene therapy could be applied to human heart attac...

Man Blind for 40 Years Regains Some Sight Through Gene Therapy

Doctors for the first time have used a form of gene therapy to restore partial vision in a blind person, according to findings announced Monday.

The research team genetically altered retinal ganglion cells to become light-sensitive in a man whose vision was destroyed by retinitis pigmentosa, a genetic disorder that breaks down cells that absorb and convert light into brain signals.

...

Gene Therapy Uses HIV to Rescue Kids Born Without Immune System

Cora Oakley is a rough-and-tumble 4-year-old who loves gymnastics and outdoor activities, particularly if it involves bouncing on a trampoline.

It's hard to tell from looking at her that she was born without an immune system. Kids with this condition can acquire dangerous, life-threatening infections from day-to-day activities as simple as going to school or playing with friends.

"I...

Early Promise for Therapy Against Duchenne Muscular Dystrophy

An experimental gene therapy for Duchenne muscular dystrophy shows promise, a small study suggests.

The severe form of muscular dystrophy -- which affects about one in 3,500 males born each year in the United States -- causes muscles to progressively weaken and lose the ability to regenerate after an injury.

Muscle tissue is eventually replaced by fat and collagen. Many children wit...

Could Gene Therapy Cure Sickle Cell Disease? Two New Studies Raise Hopes

A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients' bone marrow cells, researchers report.

Both therapies work by switching on a gene that promotes production of fetal hemoglobin, said Dr. Lewis Hsu, chief medical officer of the Sickle Cell Disease Association of America.

Sickle cell dis...

Could Gene Therapy Stem the Damage of Parkinson's?

It may be possible to protect Parkinson's patients' brains from further damage by turning off a "master regulator" gene, researchers report.

"One of the biggest challenges in treating Parkinson's, other than the lack of therapies that impede disease progression, is that the disease has already laid waste to significant portions of the brain by the time it is diagnosed," said researche...

CRISPR Gene Editing Creates 'Designer' Immune Cells That Fight Cancer

In a first, scientists have used gene-editing technology to create "designer" immune system cells that can fight tumors and survive for months in cancer patients' bodies.

It's a proof of principle, the researchers say -- and an early step toward bringing the gene-editing tool known as CRISPR into cancer treatment.

CRISPR allows researchers to precisely "snip" bits of DNA wit...

Researchers Alter Mosquitoes to Resist Dengue Infection

Mosquitoes that can't be infected by or spread dengue virus have been created by scientists.

The researchers genetically engineered the mosquitoes to be resistant to all four types of dengue, a mosquito-borne virus that's a significant global health threat.

This is the first time that mosquitoes have been genetically engineered to be resistant to all types of dengue, which c...

Gene Therapy May Be Long-Term Cure for Type of Hemophilia

A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.

Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.

...

'Designer Babies' a Long Way Off

"Designer babies" aren't going to be a reality anytime soon, researchers say.

Concerns about genetically altering embryos to have desired traits have been around nearly as long as in vitro fertilization (IVF) and the technology to screen embryos have existed.

But while recent live births resulting from embryonic CRISPR gene editing have re-focused attention on the issue, the...

How Chinese Gene-Editing Could Backfire: Babies Might Have Shorter Lives

Last year, a scientist from China created a storm of controversy when he claimed he'd used gene-editing technology to create "designer" twin babies. Now, a new study is highlighting one of the dangers of that endeavor.

Researchers have found that the gene mutation the scientist used -- affecting a gene called CCR5 -- is associated with a shorter life span: People who carry two copies ...

Gene Therapy May Help Fight Tough-to-Treat Blood Cancer

A gene therapy that tweaks the immune system might offer hope to people with blood cancer that has resisted standard treatments, a new preliminary trial suggests.

The cancer, called multiple myeloma, arises in certain white blood cells. It is currently incurable, but there are treatments that can help people live with the disease for years.

Howeve...

Gene Therapy Might Prove a Cure for 'Bubble Boy' Disease

They were once imprisoned in plastic bubbles that cut them off from the dangerous, infectious world.

But now, children born with a disease that robs them of a functioning immune system may have gained a new lease on life.

Researchers say gene therapy may be a cure for X-linked severe combined immune deficiency (SCID-X1), widely known as "bubble boy disease."

Ten ...

After Chinese Infant Gene-Editing Scandal, U.S. Health Officials Join Call for a Ban

The controversy over a Chinese scientist who claimed he created gene-edited babies has prompted the U.S. National Institutes of Health to join an international moratorium on such research.

"Today, leading scientists and ethicists from seven countries have called for an international moratorium on the use of genetic editing to modify the human germline for clinical purposes," NIH Direc...

Someday, a Pig's Heart Might Save a Child's Life

The supply of donor organs for infants needing a heart transplant is critically low, but researchers have taken a first step toward using pig hearts to fill the need.

The concept of using animal organs to save human lives has been around for years. With donor organs in short supply, the hope is that animal organs can keep patients alive while they await a human donor.

One ma...