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luxturna

Highly anticipated Luxturna gene therapy for blindness costs $850,000

The trouble had started over a decade ago, when the Hogans noticed something wasn’t right with their son Jack...When they ...
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FDA puts clinical hold on Solid Biosciences’ gene therapy trial for Duchenne muscular dystrophy over toxicity concerns

The FDA has imposed a full clinical hold on Solid Biosciences’ Phase I/II trial for its lead candidate, the Duchenne ...
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Safer and cheaper: New research in ‘nanospears’ could transform gene therapies

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules ...
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Viewpoint: GlaxoSmithKline abandonment of rare disease gene-therapy drug program because it’s not a money generator hurts children

When GlaxoSmithKline, long a global leader in the effort to pioneer gene replacement therapies, announced it would halt its drug ...
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Cryogenics logistics: Delivering CAR-T treatments at minus 240 degrees to save lives

Last year, the FDA approved the first CAR T-cell treatments—a new class of promising therapies that train the body’s immune ...
Cancer chemotherapy

Immunotherapy mystery: Drugs treat ovarian cancer ‘when they should not have’

[Four women with rare ovarian cancer], strangers to one another living in different countries, asked their doctors to try new ...
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Transforming modern medicine doesn’t have to be high tech or expensive

While intricate high-tech breakthroughs are incredible, it's important to realize the value of low-cost and low-tech ingenuity ...
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CAR-T cell therapy could drive a ‘revolution’ in cancer treatments

[T]he United States Food and Drug Administration (FDA) made the landmark decision to approve two ‘drugs’ that use CAR-T cell ...
china

Esophageal cancer treated with risky modified T-cell therapy in China

Esophageal cancer is one of the most common forms of cancer in China. Like many other types, cancer of the esophagus ...
gene

Cutting gene therapy side effects by finding a better delivery system to the brain

Researchers have found a structure on the small viruses that deliver gene therapy that makes them better at crossing from ...
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Stop AIDS? Gene therapies target ‘almost impossible to cure’ HIV

Experts say gene therapy could finally be used to treat HIV and AIDS - after a decade of trying to replicate the ...
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Video: Biohacker documents his own DIY gene therapy

DIY gene therapy is far from the mainstream, but the numbers of people who are attempting to genetically engineer their ...
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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 ...
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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 ...
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Former Novartis chief Joseph Jimenez discusses breakthroughs in gene therapy, future of CRISPR in medicine

Editor's Note: Novartis CEO Joseph Jimenez retired in January after eight years in the top job at one of the ...
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Gene therapy setback? Animal deaths in treating muscular dystrophy spark new concerns over high doses

An influential scientist involved in gene therapy’s biggest setback, the death of a study volunteer 19 years ago, has issued a ...
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Search and destroy: CAR-T cell therapy could give patients HIV immunity

A UCLA-led research team has created blood-forming stem cells that can carry a gene that allows the body to produce cells ...
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Human muscles from stem cells: Advance could aid research into muscular dystrophy, other diseases

Muscle created with stem cells is not quite as strong as the researchers would like. But they think these new ...
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China poised to do gene therapy ‘better and cheaper’ than US

China wants to go big on biotech and already is marching into experimental gene therapy. … So far, CAR-T has ...
Clinical Research

‘Supercharged’ T-cells show promise against Hodgkin’s lymphoma

Cancer has been winning the arms race against the immune system for too long, but scientists are developing plenty of ...
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Rural-urban divide: Groundbreaking gene therapies could exacerbate inequality in cancer care

Two new cancer treatments have shown miraculous cures, but if you happen to live in Arkansas or Montana, or a ...
gene therapy

Report: Price for blindness gene therapy Luxturna is ‘four times too high’ at $850,000

The $850,000 list price for a new medicine that treats a genetic form of childhood blindness is about four times too high ...
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Can gene therapy reduce terminal cancers to minor chronic diseases that are ‘no different than high blood pressure’?

On Oct. 15 at 8 a.m., Andy Lindsay stood atop 21,247-foot Mera Peak in Nepal, a wildly improbable place for ...
gene therapy

CAR-T gene therapy treatments may fall short of corrective goals, requiring surgical gene editing

For decades, researchers, some physicians, and a few patients have had visions of treatments that would go in and fix ...
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Could we fight HIV with new Car-T therapy?

The same kind of DNA tinkering that produced the first FDA-approved gene therapy for cancer has shown hints of suppressing and even ...
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Luxturna gene therapy approved for vision loss

The first two novel gene therapies for cancer treatment passed through FDA approval earlier this year, first B-cell acute lymphoblastic leukemia (ALL) followed quickly ...
gene therapy

Should patients—not the FDA— have final say on risky gene therapy treatments?

Working with mice, researchers have used gene therapy to restore sight to the blind, reprogram the body’s own T cells ...
breakthrough

Looking back at 2017’s genetics breakthroughs

It was a big year for the building blocks of life. Here were the most significant breakthroughs in genetics research ...