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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 ...
Stop AIDS? Gene therapies target 'almost impossible to cure' HIV

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 ...
Search and destroy: CAR-T cell therapy could give patients HIV immunity

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 ...
1-18-2018 Clinical Research (1)

‘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 ...
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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 ...
Can gene therapy reduce terminal cancers to minor chronic diseases that are 'no different than high blood pressure'?

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 ...
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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 ...
Could we fight HIV with new Car-T therapy?

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 ...
Luxturna gene therapy approved for vision loss

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 ...
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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 ...
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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 ...
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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 ...
FDA approves first gene therapy for a genetic disease—onetime cure for rare blindness

FDA approves first gene therapy for a genetic disease—onetime cure for rare blindness

The first gene therapy to restore sight to individuals who suffer from a rare inherited genetic blindness was approved by ...
Italian family's inability to feel pain spurs research on relief for chronic sufferers

Italian family’s inability to feel pain spurs research on relief for chronic sufferers

The six members of the family barely notice broken bones or severe burns. Researchers have identified the mutation behind their ...
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Hemophilia A gene therapy trial results: ‘This is huge’

British doctors say they have achieved "mind-blowing" results in an attempt to rid people of haemophilia A. Patients are born ...
Obesity epidemic: Could gene therapy be an answer?

Obesity epidemic: Could gene therapy be an answer?

Scientists are working with treatments that have shown success in amping up the metabolism of mice, helping them lose weight ...
Cancer chemotherapy

Cancer treatment without chemotherapy? CAR T-cells could become the new standard

Although rare, it is not unheard-of for new treatments to achieve substantial early successes in one or two patients only ...
2017: The year gene therapy became a 'clinical reality'

2017: The year gene therapy became a ‘clinical reality’

This year, gene therapy finally became a clinical reality. The U.S. Food and Drug Administration approved two personalized treatments that ...
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Scientists sound alarm over DIY gene therapy, calling it ‘unsafe’

In just the past few months we’ve seen not one, but two examples of people pursuing unregulated gene therapy. … [T]he largest organization of ...
Gene therapy, CRISPR could provide treatments for sickle-cell, but cures far off

Gene therapy, CRISPR could provide treatments for sickle-cell, but cures far off

More than 50 years after the cause of sickle-cell disease was discovered, a dozen treatments for the painful and life-shortening inherited ...