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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...

Gene Tweaking Prevented Obesity in Mice

A modified version of the CRISPR gene-editing technique could help fight obesity without having to alter any genes, a new study in mice suggests.

CRISPR, an acronym for a family of DNA sequences, generally involves cutting or editing DNA to correct genetic defects that cause disease.

This new technique boosts the activity of certain genes and prevented severe obesity in mice g...

Gene Therapy for Sickle Cell Takes Another Step Forward

A new gene therapy shows early promise against sickle cell anemia, researchers say.

The therapy targets the genetic flaw that causes sickle cell. In a small group of patients, researchers said the therapy appears safe and effective enough to keep moving forward into larger trials.

"We've been talking about using gene therapy for sickle cell anemia for years. Now it's finally...

Parkinson's Gene Therapy Wires New Brain Circuits

An experimental gene therapy for Parkinson's disease seems to work by rewiring key areas of the brain, a new study finds.

The researchers focused on 15 Parkinson's patients who, in an earlier trial, had received so-called GAD gene therapy. GAD is an enzyme that spurs the production of a brain chemical involved in movement control.

In the previous trial, patients had shown im...

Gene Therapy for Parkinson's Symptoms Shows Promise

A new gene therapy might help improve motor symptoms in people with Parkinson's disease who aren't responding to other therapies, an early study has found.

"This is not a cure of Parkinson's disease," said James Beck, chief scientific officer of the Parkinson's Foundation. "This is a potentially good treatment for symptom control. It provides an additional way of providing dopamine t...

Gene 'Editing' in Dog Study Shows Promise for Kids With Muscular Dystrophy

For the first time, scientists report using gene-editing technology to halt the progression of muscular dystrophy in dogs -- suggesting a possible breakthrough for children with a form of the disease.

Reporting in the current issue of Science, researchers describe how they used CRISPR technology to edit a naturally occurring genetic flaw that causes a version of Duchenne muscul...

Americans Say Editing Baby's Genes OK, Up to a Point: Poll

Gene editing is no longer just fodder for sci-fi movies. Most Americans believe it's OK to tweak a baby's DNA under certain circumstances, a new poll finds.

The Pew Research Center, based in Washington, D.C., found almost three-quarters of Americans approve of gene editing to prevent serious health problems in babies.

But tampering with their intelligence is going too far, ...