FDA

Seeking a new path for FDA regulation of revolutionary medical treatments

Researchers are developing therapies that could permanently alter a patient’s genes... . The early results are promising. Patients would live ...
gene therapy

Bringing us closer to ‘once-in-a-lifetime’ hemophilia treatment with experimental gene therapies

Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. After ...
gene therapy

Mainstream medicine? US health officials want to ease restrictions on gene therapy experiments

U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly ...
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Examining the legacy of W. French Anderson—’father of gene therapy’ and convicted child molester

[Dr. W. French] Anderson has been hailed as the father of gene therapy and was honored at George H.W. Bush’s ...
dmd

Duchenne muscular dystrophy gene therapy trial put on hold

Sarepta Therapeutics’ clinical trial for a gene therapy to treat Duchenne muscular dystrophy has been paused by the Food and ...
gene therapy womb

Are we close to gene therapy in the womb?

[Recently] scientists reported that they were able to treat a serious genetic disorder in the womb — in mice. It sounds like ...
PRinc photo of fetus at weeks

Deadly brain disease could be treated in the womb with gene therapy

A research team from the UK and Singapore showed that a neurodegenerative condition called Gaucher disease, which can be fatal, ...
enhance

Viewpoint: We’re ‘nowhere close’ to being ready to edit human genomes

Genome meddling to cure diseases is often worth the risk, but nothing else is just yet ...
space travel

How gene therapy could help astronauts survive deep space deadly radiation

Over the past five decades, space travel advocates have been pushing to expand our footprint in space. They dream about lunar ...
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Who should pay for million-dollar life-saving gene therapies?

While life-changing and life-saving gene therapies are going on the market, they have price tags that many cannot pay for ...
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Money magnets: Wall Street enamored by promise of human gene editing, gene therapies

Venture capitalists and investors are pouring money into the genomics sector, seeking to capitalize on breakthroughs in CRISPR gene editing ...
CRISPR

What’s stopping us from using CRISPR to gene edit humans to fight disease?

Emerging clinical applications of CRISPR editing include delivery of CRISPR systems into the body to repair genetic sequences. This is ...
Trends GenderBreakdown px x

Gene-editing advances put us at the dawn of a revolution in medicine

So much of the public's attention is focused on the use of genetic engineering in the crops that our farmers ...
knees

Incurable arthritic knees? Gene therapy offers new hope

Imagine a single injection into the knee vanquishing painful arthritis — forever. Gene therapies aimed at two molecular targets could ...
gene editing

Should it matter if the public is wary of gene editing and human enhancement?

A survey of people in the US and 10 European nations found opposition to some forms of gene editing for ...
gene therapy

Gene therapy challenge: How much should it cost and how do we pay for it?

When evaluating the high prices of one-time gene therapy treatments, should we look to the costs of organ transplants when ...
dystrophy

Talking Biotech: Pet dogs with genetic diseases testing ground for gene therapy

Journalist Emily Mullin: Gene therapies becoming more promising but restricted in humans because of safety concerns, prompting some researchers to ...
stemcells

Gene therapy creates boy’s replacement skin from his stem cells

Doctors treated a 7-year-old boy’s devastating genetic skin disease—junctional epidermolysis bullosa—by genetically modifying his stem cells to create a new ...
beers

Upchuck factor: Alcoholism targeted with novel gene therapy

Researchers are exploring the use of a naturally occurring mutation that causes people to get sick after drinking alcohol. The ...
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Could gene editing have saved Charlie Gard?

The British infant died of a mitochondrial disease as news broke that US scientists had successfully edited a human embryo ...
blindness

Pioneering gene therapy restores vision to people with rare retinal blindness

Spark Therapeutics is moving forward with its treatment for a type of inherited retinal dystrophy. In the wake of the ...
hiv

HIV fix: Can gene editing work alongside the virus to provide a cure?

HIV is no longer a death sentence in much of the developed world. But effective treatment in developing nations needs ...
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2 young leukemia patients cured with T-cell immunotherapy using genetically engineered donor cells

Gene-edited immune cells cure two British babies with childhood leukemia. But instead of patient-specific cells, doctors used cheaper universal cells ...
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Genetics for public health: A lost cause?

Its power for discovering rare diseases is well proven. But genetics’ impact on public and preventative health has a long ...
gene editing

CRISPR-Cas9 is hot but it’s not the only way to edit a genome

Despite its growing track record and huge potential, CRISPR-Cas9 genome editing may not be the magic bullet for all applications ...
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After early setbacks, gene therapy’s comeback nearly complete

After some horrifying early setbacks, gene therapy's back. Researchers have learned from early mistakes to make the therapy safer and ...
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Genetic therapies won’t solve depression anytime soon

Can a better understanding of the genetics of depression lead us to safer, more effective treatments for the disease? ...
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Gene doping in sports entails challenges and dangers but may not be so dopey

Athletes experimenting with gene doping may not be ethical, but it could lead to potential treatments of human disease ...
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