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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 ...
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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 ...
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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 ...
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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 ...
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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 ...
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Transplanting stem cells directly into brain could alter treatment of nervous system disorders

A therapeutic technique to transplant blood-forming (hematopoietic) stem cells directly into the brain could herald a revolution in our approach ...
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Gene therapy challenge: How much should it cost and how do we pay for it?

When evaluating the high prices of one-time gene therapy treatments, should we look to the costs of organ transplants when ...
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Fighting infectious diseases with immunotherapy on ‘cusp of commercialization’

Immunotherapy, which involves adapting immune cells to destroy specific cellular targets, has made a name for itself treating cancer. But ...
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Gene therapy could be ‘ideal cure’ for hemophilia B

[L]ast year, [Jay] Konduros enrolled in a clinical trial, receiving an experimental gene therapy at Children's Hospital of Philadelphia [to ...
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DIY gene therapy? FDA is not a fan

A few biohackers (fans of do-it-yourself (DIY) science experiments) recently designed a gene therapy for HIV. Not only that, they posted a video ...
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Exorbitant costs of gene therapy raise payment concerns for patients and government

[Editor's note: Executives from several companies developing gene therapies gathered for a panel discussion at the recent Forbes Healthcare Summit, ...
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Genetic engineering, synthetic biology poised to boost photosynthesis and carbon capture

In a feature article published in the open access journal eLife, an international team of experts led by Dr Bonnie Wintle ...
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Can we solve the patent issues that threaten CRISPR research?

[Editor's note: Lawrence Horn is President and CEO of MPEG LA, which provides licenses for standards and other technology platforms.] Although recent ...
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Insecticide resistance threatens to derail malaria control in Africa

The largest genetic study of mosquitoes has found their ability to resist insecticides is evolving rapidly and spreading across Africa, ...
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Why life-saving gene therapy isn’t available yet to children who need it most

[The first patient to permanently edit his DNA,] Brian Madeux, 44, of Arizona, is part of a clinical trial testing ...
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Gene therapy challenge: Overcoming shortage of key and expensive viruses

Eager to speed development of revolutionary treatments, the Food and Drug Administration recently announced that it would expedite approval of ...
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How gene therapy could help fight methamphetamine addiction

Gene therapy, which modifies a person’s DNA, has long been thought of as a way to treat genetic diseases—and, more recently, ...
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Exploring national security risks related to gene editing

Technology is advancing exponentially and the exciting field of genome editing is no exception. Scientists at the University of Wisconsin-Madison ...
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Viewpoint: Cost of gene therapy could put experimental treatments out of reach for most

Drugs that modify human genes have the potential to cure intractable diseases with just one treatment. Few could disagree that's ...
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Canadian researchers can’t use CRISPR in human embryo research

In the United States, using genetic engineering techniques such as CRISPR to make genetic alterations that can be passed on ...
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FDA details risk-based cellular therapy and regenerative medicine guidelines

The FDA has issued two final guidances and two draft guidances, all designed to articulate the agency’s approach to developing ...
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Talking Biotech: Pet dogs with genetic diseases testing ground for gene therapy

Journalist Emily Mullin: Gene therapies becoming more promising but restricted in humans because of safety concerns, prompting some researchers to ...
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New T-cell treatment shows promise for treating leukemia

A new way of genetically altering a patient’s cells to fight cancer has helped desperately ill people with leukemia when ...
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Gene therapy boost: FDA positions for faster reviews of new treatments

The Food and Drug Administration on [November 16] issued new guidelines to speed the introduction of treatments involving human cells ...
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FDA likely to approve hereditary blindness gene therapy

[A]nother gene therapy is on the cusp of approval, this time to treat a form of hereditary blindness. If given ...
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A gene-editing first: Scientists try to edit a living human’s DNA

Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change ...