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Gene therapy could restore color vision for people who see the world in shades of gray

In a small trial in Germany, an experimental gene therapy improved the vision of nine people with total color blindness, ...
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Ultimate fitness hack: Imagine being able to build muscles with a gene therapy

Trying to hack fitness is a multi-million-dollar industry; we’ve all seen at least one ad featuring a purported miracle product ...
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What’s holding up gene therapy? Making drugs on a large scale

As interest in gene therapy grows, manufacturers face physical, biological, and engineering challenges to developing and producing drugs on a ...
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Podcast: Fighting blindness with CRISPR. Ophthalmologist in groundbreaking study explains how gene editing could treat a once-incurable disease

Congenital eye disorders can rob children of their eyesight at a young age and severely diminish their quality of life ...
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Common form of inherited blindness shows ‘massive improvements’ with experimental gene therapy

This month, K.L. became one of the first patients to receive a new experimental gene therapy for children with a ...
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Building ‘better’ astronauts through genetic engineering could be key to colonizing other planets

Through genetic engineering, we will one day have the ability to thrive in harsh alien environments ...
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Gene therapy shows success against some cancers and inherited disorders. Can it tackle obesity?

Scientists are working with treatments that have shown success in amping up the metabolism of mice, helping them lose weight ...
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900 gene therapy drugs are in the pipeline. How does the FDA want to regulate them?

To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The ...
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Gene therapy for Alzheimer’s? Biotech startup suggests new approach, using dementia ‘off switch’

What if there were a single mechanism in the brain that, when faulty, leads to all kinds of dementias? And ...
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Teenager’s experimental gene therapy treatment could change the lives of millions of sickle cell patients worldwide

Meet Helen Obando, a shy 16-year-old who likes to dance when her body isn’t ravaged by the debilitating symptoms of ...
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Gene therapy era: A look at current treatments and what’s next for cancers and genetic disorders

At least nine gene therapies have been approved for certain kinds of cancer, some viral infections and a few inherited ...
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Gene therapy offers hope for untreatable CTE brain disorder

[Researchers from Weill Cornell Medical College] published a new study (“Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy”) in Human Gene Therapy that ...
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Virus used to deliver gene therapy treatments may increase liver cancer risk, study suggests

Just as gene therapy finally seems to be living up to its promise, a study has revived a lingering worry ...
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Forecasting: 2020 could bring major advances in gene therapies and pig-to-human organ transplants

Here are two stories in biotech we’re keeping our eyes on. Although successes likely won’t come to fruition this year ...
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Will 2020 see the debut of promising gene therapy for hemophilia A? It’s up to the FDA.

Hemophilia A gene therapy has been twenty years in the making ...
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2019 offered ‘eclectic’ mix of potentially breakthrough treatments for genetic diseases, from cystic fibrosis to sickle cell

2019 was a very good year for new treatments of genetic diseases ...
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Would price controls hamper research on gene therapy and other innovative treatments?

Over the next decade, it is a near certainty that we will have gene-therapy cures for deadly inherited disorders such ...
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$1 million for anti-aging gene therapy? ‘Dubious’ treatment raises ethical concerns

Would you pay $1 million and fly to South America for a chance to live longer? Libella Gene Therapeutics, a ...
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Viewpoint: Spinal muscular atrophy treatment Zolgensma a gene therapy trailblazer, despite controversy

In August, the US Food and Drug Administration (FDA) gave drug manufacturer AveXis, Inc, a subsidiary of Novartis AG, a ...
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Viewpoint: Experimental gene therapy saved my sons’ eyesight

Not long after we brought my newborn son, Anthony, home from the hospital, we noticed his eyes kept darting to ...
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Gene therapy may offer cure for debilitating ‘bubble boy disease’

Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the “bubble boy ...
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Trio of gene therapies seeks to reverse age-related diseases to make us ‘healthy, youthful later in life’

The legendary synthetic biologist Dr. George Church and team at the Wyss Institute at Harvard University took a first step towards ...
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Expensive CAR-T therapy raises difficult question: ‘What is the price of a human life?’

A new type of therapy for certain blood cancers is ingenious but it raises important questions about its cost, both ...
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Patient’s ‘serious’ kidney injury halts gene therapy trial for Duchenne muscular dystrophy

The Food and Drug Administration has halted a clinical trial involving a Duchenne muscular dystrophy gene therapy from Solid Biosciences ...
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Rejuvenate Bio’s gene therapy shows promise at reversing aging—in mice

Harvard synthetic biology pioneer George Church generated some buzz last year when he co-founded Rejuvenate Bio with the goal of ...
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Viewpoint: Netflix’s new horror movie ‘Eli’ is a fright. But why did they have to ‘tarnish gene therapy’?

Attempts to treat genetic disease shouldn’t be the stuff of horror films ...
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Gene therapy pioneer James Wilson makes a comeback after disastrous start

Twenty years ago, a young man named Jesse Gelsinger was injected with a large dose of gene-shuttling viruses designed in ...
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Gene editing used in China in a failed but promising attempt to cure HIV

Scientists in China have used CRISPR gene-editing technology to treat a patient with HIV, but it didn't cure the patient, ...