7-26-2018 ANDERSON

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 ...
7-16-2018 cells_16x9

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 ...
7-1-2018 Immunotherapy

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 ...
6-25-2018 DSC_4748-760x350

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 ...
4-1-2018 7c01c6_b123a9cdfd4c4059b63fcbc9302f6d09_mv2

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 ...
5-28-2018 puppysmile

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 ...
5-21-2018 JackHogan_eye

First Luxterna results: Does the $850,000 gene therapy work?

About eight weeks earlier, [Jack Hogan had] been the first person to get an $850,000 therapy called Luxturna since it had hit the market ...
Can we reverse aging in dogs through gene therapy? If so, humans could be next.

Can we reverse aging in dogs through gene therapy? If so, humans could be next.

The world’s most influential synthetic biologist is behind a new company that plans to rejuvenate dogs using gene therapy. If ...
5-9-2018 Amish Clinic

Paying for pricey gene therapy without insurance: Amish community faces challenge

[A]t $850,000 a person, Luxturna was more budget-busting than just about any other drug. [Company] Spark had proposed a few different ways of helping ...
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Death of controversial biohacker Aaron Traywick puts movement at ‘crossroads’

At just 28, [Aaron] Traywick was among the most infamous figures in the world of biohacking—the grandiose CEO of a ...
5-3-2018 evewkellys0379

Gene therapy research gets boost from parent-led crowdfunding

It’s a compelling narrative: A parent learns that his or her child has a fatal disease with no cure, and, ...
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Beta thalassemia patients could be freed from life of blood transfusions by one-time gene therapy

A one-time, experimental treatment for an inherited blood disorder has shown dramatic results in a small study. It’s a major ...
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Pediatric brainstem cancer eradicated in mice using CAR-T treatment

Engineered human immune cells can vanquish a deadly pediatric brain tumor in a mouse model, a study from the Stanford University ...
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Gene therapy and rare diseases: ‘Is curing patients a sustainable business model?’

An analyst at Goldman Sachs asked a troubling question...about gene therapy. “Is curing patients a sustainable business model?” In social media, reactions ...
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Travel to Mars: Can gene therapy help us clear deadly radiation hurdle?

An international group of researchers has come up with a new plan to help astronauts survive high-level radiation in space ...
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Pursuing ‘organic gene therapy’ for sickle cell anemia and beta thalassemia

Scientists in Australia have solved a 50-year-old mystery that could lead to the development of new gene therapies for blood ...
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Money magnets: Wall Street enamored by promise of human gene editing, gene therapies

Venture capitalists and investors are pouring money into the genomics sector, seeking to capitalize on breakthroughs in CRISPR gene editing ...
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House ‘right to try’ law passes, sent to Senate: What it could mean for fragile patients

The US House of Representatives passed a federal "right-to-try" bill [March 21], leaving many Americans wondering what the move could ...
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Highly anticipated Luxturna gene therapy for blindness costs $850,000

The trouble had started over a decade ago, when the Hogans noticed something wasn’t right with their son Jack...When they ...