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Resurgence of gene therapy has dramatically altered the the biomedicine revolution

Some technologies that have emerged and altered the landscape in recent years include immunotherapy, CRISPR-Cas9 gene editing, and chimeric antigen ...
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‘Gigantic leaps forward’ What’s happened in the 21 years since a teenager died undergoing gene therapy, stalling emergence of this biomedical breakthrough?

In 1999, [teenager] Jesse [Gelsinger] received a dose of the ornithine transcarbamylase gene, engineered into a recombinant adenovirus, at the ...
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Podcast: Rare genetic disorders and pregnancy—Navigating an ’emotionally challenging’ journey

We look at the progress that’s been made in tackling rare genetic disorders (and the challenges that remain) and we ...
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Herpes cure? Gene therapy may work

Infectious disease researchers at Fred Hutchinson Cancer Research Center have used a gene editing approach to remove latent herpes simplex ...
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Gene therapy for hemophilia delayed until 2022 after FDA rejects one-time treatment, shocking doctors and scientists

U.S. regulators rejected [Biomarin’s] potentially game-changing hemophilia A gene therapy over concerns it might not really be a one-and-done lifetime ...
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One year in, 9 boys with muscular dystrophy show remarkable progress from gene therapy

Nine boys aged 6 to 12 who have been living with [Duchenne Muscular Dystrophy] since birth received a gene therapy ...
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Strokes often deadly but gene therapy offers hope to rebuild critically damaged brain cells

When you think of a typical meal in the US South, you will likely recall the rich, hearty dishes that ...
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Lucy may not live to see her second birthday without expensive gene therapy

Lucy Van Doormaal was born on April 1 in Vancouver, but before her parents, Scott and Laura Van Doormaal, could ...
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Another COVID-19 casualty: ‘Major delays’ for gene therapy trials for rare diseases

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has ...
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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 ...