They were born without a working germ-fighting system, every infection a threat to their lives. Now eight babies with "bubble boy disease" have had it fixed by a gene therapy made from one of the...
Researchers win prize for restoring children’s eyesight
LISBON, Portugal (AP) — Researchers in the United States and Britain are sharing a 1 million-euro ($1.16 million) prize from a Portuguese scientific foundation for developing revolutionary gene therapy that has restored the sight of children.
The Lisbon, Portugal-based Champalimaud Foundation's annual Vision Award was given Tuesday for work on the treatment of children blinded from birth by genetic disease.
Officials remove special rules for gene therapy experiments
U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly becoming an established form of medical care with no extraordinary risks.
MADISON, Wis. (AP) — As a doctor injected cloudy fluid into Makena Eighmy's intravenous line, it looked like the 13-year-old animal lover from near Watertown was getting a standard procedure for her cancer.
But the infusion this month at UW Health's American Family Children's Hospital was far from routine.
Inherited eye disease sees first FDA-approved gene therapy in Luxturna
Inherited eye disease sees first FDA-approved gene therapy in Luxturna
CLEVELAND, Ohio -- Allison Corona remembers every detail about the night in 2005 when she discovered that there might be a cure for her blindness.
She was up late doing research on her computer – with the screen adjusted to show large print – when she found information on Luxturna, a proposed gene therapy for one type of inherited retinal disease. That's the condition that caused Corona's severely limited sight at birth.
Can gene therapy be harnessed to fight the AIDS virus?
For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that made this possible.
Scientists removed some of his blood cells, disabled a gene to help them resist HIV, and returned these "edited" cells to him in 2014. So far, it has given the San Francisco man the next best thing to a cure.
Scientists Use Gene Therapy to Fight HIV
2nd man has gene editing; therapy has no safety flags so far
A second patient has been treated in a historic gene editing study in California, and no major side effects or safety issues have emerged from the first man's treatment nearly three months ago, doctors revealed Tuesday.
Gene editing is a more precise way to do gene therapy, and aims to permanently change someone's DNA to try to cure a disease.
Price tag on gene therapy for rare form of blindness: $850K
WASHINGTON (AP) — A first-of-its kind genetic treatment for blindness will cost $850,000 per patient, making it one of the most expensive medicines in the world and raising questions about the affordability of a coming wave of similar gene-targeting therapies.
In a milestone year, gene therapy finds a place in medicine
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the biblical-sounding feat of helping the blind to see were approved by U.S. regulators, establishing gene manipulation as a new mode of medicine.
Gene therapy for rare form of blindness wins US approval
WASHINGTON (AP) — U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major advance for the emerging field of genetic medicine.
Designer proteins that package genetic material could help deliver gene therapy
(The Conversation is an independent and nonprofit source of news, analysis and commentary from academic experts.)
Ian Haydon, University of Washington
(THE CONVERSATION) If you’ve ever bought a new iPhone, you’ve experienced good packaging.
Gene therapy shows promise against blood-clotting disease
Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood disease.
Hemophilia almost always strikes males and is caused by lack of a gene that makes a protein needed for blood to clot. Small cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding.
University of Minnesota research was pivotal in landmark gene editing trial
A bold attempt this month to cure a lethal genetic disease by editing DNA inside a patient was carried out in San Francisco and made headlines around the country but it was based largely on mouse research at the University of Minnesota Medical Center.
Now, the second, third and additional attempts may occur in Minneapolis as researchers race to prove the safety and effectiveness of editing genes inside patients.
Boy with rare disease gets brand new skin with gene therapy
LONDON (AP) — Doctors treating a critically ill boy with a devastating skin disease used experimental gene therapy to create an entirely new skin for most of his body in a desperate attempt to save his life.
Two years later, the doctors report the boy is doing so well that he doesn't need any medication, is back in school and even playing soccer.
Abeona Therapeutics announces new gene therapy facility, stock offering
Abeona Therapeutics announces new gene therapy facility, stock offering
CLEVELAND, Ohio -- Like a World Series team on a streak, Cleveland-based Abeona Therapeutics Inc. got hot in October.
Abeona Therapeutics is a clinical-stage biopharmaceutical company developing gene and cell therapies for life-threatening rare genetic diseases. Its strategy is to re-engineer viruses – which usually spread disease – to instead save lives.
‘Molly is a success story’: Gene therapy that improved Nebraska teen’s vision could soon be available nationwide
OMAHA, Neb. — The first new thing Molly Troxel saw was a familiar sight — some might even say an old friend — to most other people.
“I saw the moon,” she said. “I’d never seen it before.”
Then came more firsts: her dog’s eyelashes; the twinkle lights in the boutique next door to the pizza shop.
‘Molly is a success story’: Gene therapy that improved Omaha teen’s vision could soon be available nationwide
The first new thing Molly Troxel saw was a familiar sight — some might even say an old friend — to most other people.
“I saw the moon,” she said. “I’d never seen it before.”
Then came more firsts: her dog’s eyelashes; the twinkle lights in the boutique next door to the pizza shop.
US regulators approve 2nd gene therapy for blood cancer
TRENTON, N.J. (AP) — U.S. regulators on Wednesday approved a second gene therapy for a blood cancer, a one-time, custom-made treatment for aggressive lymphoma in adults.
The Food and Drug Administration allowed sales of the treatment from Kite Pharma. It uses the same technology, called CAR-T, as the first gene therapy approved in the U.S. in August, a treatment for childhood leukemia from Novartis Pharmaceuticals.
FDA advisers endorse gene therapy to treat form of blindness
SILVER SPRING, Md. (AP) — A potentially groundbreaking treatment for a rare form of blindness moved one step closer to U.S. approval Thursday, as federal health advisers endorsed the experimental gene therapy for patients with an inherited condition that gradually destroys eyesight.
The panel experts to the Food and Drug Administration voted unanimously in favor of Spark Therapeutics' injectable therapy, which aims to improve vision by replacing a defective gene needed to process light.
The Latest: FDA advisers endorse gene therapy for blindness
SILVER SPRING, Md. (AP) — The Latest on an FDA panel's discussion on a gene therapy treatment for blindness (all times local):
3:19 p.m.
U.S. health advisers have endorsed an experimental approach to treating inherited blindness, setting the stage for the likely approval of an innovative new genetic medicine.
Correction: Genetic Frontiers-Blindness-Gene Therapy story
In a story Oct. 9 about an experimental gene therapy for blindness, The Associated Press erroneously reported the deadline for a decision by the U.S. Food and Drug Administration. The date is Jan. 12, not Jan. 18.
A corrected version of the story is below:
Seeing hope: FDA panel considers gene therapy for blindness
FDA Panel Considers Gene Therapy For Blindness
US clears breakthrough gene therapy for childhood leukemia
WASHINGTON (AP) — Opening a new era in cancer care, U.S. health officials have approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.
US clears breakthrough gene therapy for childhood leukemia
WASHINGTON (AP) — Opening a new era in cancer care, U.S. health officials on Wednesday approved a breakthrough treatment that genetically engineers patients' own blood cells into an army of assassins to seek and destroy childhood leukemia.
The Food and Drug Administration called the approval historic, the first gene therapy to hit the U.S. market. Made from scratch for every patient, it's one of a wave of "living drugs" under development to fight additional blood cancers and other tumors, too.
A treatment for a common childhood blood cancer could become the first gene therapy available in the U.S.
A Food and Drug Administration advisory panel voted 10-0 on Wednesday in favor of the leukemia treatment developed by the University of Pennsylvania and Novartis Corp. The FDA usually follows recommendations from its expert panels, but isn't obligated to do so.