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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, ...
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A businessman’s daughter has an ‘ultra-rare’ disease, so he immersed himself in science to find a gene therapy cure

Simon and Nina Frost had spared no expense, taking Annabel to all the best neurologists around the country. Finally a ...
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We can identify ‘bad’ genes. Why can’t we use CRISPR gene editing to get rid of them?

Gene therapy 2.0 using CRISPR to knock out harmful genes sounds is a great idea--if and when it works. But ...
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Powerful CAR-T duo uses artificial ‘claws’ to grab and destroy HIV

Lengtigen, a biotech company based in Gaithersburg, MD, teamed up with researchers at the Albert Einstein College of Medicine in ...
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Application for pricey gene therapy Zolgensma contained manipulated data, but drug will remain on market, FDA says

The Food and Drug Administration said Tuesday [August 7]  that Novartis submitted manipulated data as part of its application for ...
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5 ways we could pay for expensive life-saving gene therapies

We have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ...
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$2.1 million gene therapy is most expensive drug in history

The federal Food and Drug Administration has approved a gene therapy for a rare childhood disorder that is now the ...
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How this promising gene therapy for a rare neuromuscular disease was fueled by passionate parents and a dog

The gene therapy will be too late for Joshua Frase. But it will be his legacy ...
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Why we shouldn’t view gene therapy as a treatment of last resort

Providers and payers must ask themselves a two-pronged question about gene therapy: Who should be treated and when? … The ...