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2-19-2019 angelman clinic feature

Rare brain disorder—Angelman syndrome—could be treated with gene therapy in the womb

Scientists are developing a radical form of gene therapy that could cure a devastating medical disorder by mending mutations in the brains ...
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First human test for gene therapy targeting most common cause of blindness

A woman from Oxford has become the first person in the world to have gene therapy to try to halt ...
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First gene-editing experiments on adults suffering from rare metabolic disorders offer promising early results

In late 2017, scientists first began attempting to edit the genes of adults to treat rare genetic disorders. Preliminary results ...
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Chinese scientists aren’t keeping tabs on experimental gene therapy patients, report says

Gene therapies are very much at their preliminary stages of development, so it would make sense to keep tabs on ...
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Can gene therapy offer a cure for sickle-cell disease?

[I]n November, six months after [21-year-old Manny] Johnson became the first patient to receive an experimental therapy aimed at curing ...
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Inside a couple’s quest to pay for an experimental gene therapy to save their children

“We need your help, we really do,” Gary [Landsman] says, his voice breaking. The Landsmans’ two sons—Benny, then 18 months, ...
Gene therapy for autism? Rare Angelman syndrome could answer key questions

Gene therapy for autism? Rare Angelman syndrome could answer key questions

[T]he success of genetic therapies [for autism] is not guaranteed. Even if disrupted genes can be replaced, repaired or otherwise ...
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CAR-T cell therapy and the promise of immune cells engineered to fight cancer

The National Cancer Institute estimates that more than 1.7 million people will be diagnosed with cancer this year. To cope ...
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Can we afford gene therapy’s million-dollar price tags?

[4-year-old] Caspian was born with a rare, inherited eye disorder called Leber congenital amaurosis, which results in the progressive deterioration ...
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Were ‘crucial contributors’ snubbed in awarding Nobel prize for cancer immunotherapy?

[I]t’s rare that Nobel announcements don’t produce grumblings about who was left out, and this year was no exception. At ...
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Seeking a new path for FDA regulation of revolutionary medical treatments

Researchers are developing therapies that could permanently alter a patient’s genes... . The early results are promising. Patients would live ...
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Bringing us closer to ‘once-in-a-lifetime’ hemophilia treatment with experimental gene therapies

Scientists are edging closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots. After ...
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Mainstream medicine? US health officials want to ease restrictions on gene therapy experiments

U.S. health officials are eliminating special regulations for gene therapy experiments, saying that what was once exotic science is quickly ...
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Examining the legacy of W. French Anderson—’father of gene therapy’ and convicted child molester

[Dr. W. French] Anderson has been hailed as the father of gene therapy and was honored at George H.W. Bush’s ...
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Duchenne muscular dystrophy gene therapy trial put on hold

Sarepta Therapeutics’ clinical trial for a gene therapy to treat Duchenne muscular dystrophy has been paused by the Food and ...
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Are we close to gene therapy in the womb?

[Recently] scientists reported that they were able to treat a serious genetic disorder in the womb — in mice. It sounds like ...
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Deadly brain disease could be treated in the womb with gene therapy

A research team from the UK and Singapore showed that a neurodegenerative condition called Gaucher disease, which can be fatal, ...
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Viewpoint: We’re ‘nowhere close’ to being ready to edit human genomes

Genome meddling to cure diseases is often worth the risk, but nothing else is just yet ...
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How gene therapy could help astronauts survive deep space deadly radiation

Over the past five decades, space travel advocates have been pushing to expand our footprint in space. They dream about lunar ...
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Turning white blood cells into ‘living cancer drugs’ with electric shocks

A promising new class of cancer treatments recruits the cells in our blood to fight tumors, using powerful gene-editing tools ...
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Making CAR-T cancer treatments less risky with ‘safety switches’ and precision targeting

A majority of patients who receive CAR-T cell therapy react [with] varying degrees of severity. Those same T cells that ...
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Muscular dystrophy targeted with promising gene therapy

An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne ...
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Key question facing new gene therapy for blood disorders: How long will it last?

New versions of Bluebird Bio’s gene therapy for inherited blood disorders yielded significant benefits for patients, according to updated results from ...
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Is gene therapy the answer to spinal cord injuries?

Here’s the thing about the spinal cord: you’ve only got one. And right now, if you injure it, doctors can’t ...
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Expanded genetic testing of newborns could help us get the most out of new gene therapies

The company behind [spinal muscular atrophy drug] AveXis, is seeing the most dramatic results in children who are treated in ...
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Who should pay for million-dollar life-saving gene therapies?

While life-changing and life-saving gene therapies are going on the market, they have price tags that many cannot pay for ...
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Quick fix: Gene therapy could offer less invasive pet contraception

Might gene therapy provide a one-time, far less invasive way to ensure that cats and dogs do not beget kittens ...
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Using patient registries to track effectiveness of cell and gene therapy trials

Due to advances in rare-disease research and individualized cell and gene therapies, there has been a recent crop of treatments ...