Two new studies using CRISPR gene editing offer potential new treatments for Alzheimer's disease.

"A pipeline of potential new treatments offers hope for the Alzheimer's and dementia community,"said Maria Carrillo, chief science officer for the Alzheimer's Association. "The progress and approvals we...

A clinical trial that's attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients.

Two of the patients were treated at Cleveland Clinic Children's in Ohio, and doctors there are hopeful that their positive results will be borne out in future research.

"New treatments like this are critical for people who have sickle...

Millions of stray cats roam the world over, and surgical sterilization has long been the primary method of population control.

But a small new study shows promising results for a one-and-done contraceptive injection.

Researchers say this first-of-its-kind approach appears safe and effective.

"A non-surgical contraceptive that could result in lifetime sterility following a...

An exceptionally pricey gene therapy cure for sickle cell disease could soon be available, but it's not clear whether insurance companies will balk at the cost and deny coverage.

On the surface, the gene therapy does not appear as cost-effective as the grinding medical care that sickle cell patients now receive, according to a new analysis.

Gene therapy applied just once to a sickle...

"Zinc fingers"might sound like the world's worst candy bar, but these human proteins might prove key to treating complex genetically driven diseases.

A new artificial intelligence program is poised to enable the simple production of zinc fingers, according to research co-led by the N...

Ten children with an especially rare and hard-to-treat form of "bubble boy" disease are living normal lives after receiving a new gene therapy approach, researchers say.

Experts said the findings are a major advance for children with the disease -- a subtype of severe combined immunodeficiency (SCID).

SCID refers to a group of rare genetic diseases that cause babies to be born ...

Patients with a high-risk bladder cancer now have a new option to treat it.

The U.S. Food and Drug Administration on Friday approved a gene therapy called Adstiladrin, which is designed to work for patients who have what's called high-risk non-muscle-invasive bladder cancer (NMIBC) that hasn't responded to the standard treatment, Bacillus Calmette-Guérin (BCG), but hasn't spread. BCG i...

An experimental gene therapy that's applied as a skin gel appears to heal wounds caused by a rare and severe genetic skin disease.

Experts called the findings "remarkable," and said they bring hope of a better quality of life to children and young adults living with the condition, called dystrophic epidermolysis bullosa (DEB).

The disease affects about 3 out of every 1 million peopl...

People with one form of the genetic blood disorder hemophilia now have a one-time treatment with a $3.5 million price tag.

The U.S. Food and Drug Administration approved the new gene therapy Hemgenix on Nov. 22. Soon after, drugmaker CSL Behring revealed its cost.

The company said its drug would ultimately reduce health care costs because patients with the genetic disorder would ne...

Doctors are hopeful that an innovative treatment performed before birth may help children born with the rare genetic, and often fatal, condition called Pompe disease.

A thriving Canadian toddler is evidence that treatment while still in the womb offers better outcomes.

Doctors from the United States and Canada published a case study Nov. 9 in the