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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 ...
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 ...
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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 ...
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FDA puts clinical hold on Solid Biosciences’ gene therapy trial for Duchenne muscular dystrophy over toxicity concerns

The FDA has imposed a full clinical hold on Solid Biosciences’ Phase I/II trial for its lead candidate, the Duchenne ...
Safer and cheaper: New research in 'nanospears' could transform gene therapies

Safer and cheaper: New research in ‘nanospears’ could transform gene therapies

UCLA scientists have developed a new method that utilizes microscopic splinter-like structures called “nanospears” for the targeted delivery of biomolecules ...
3-10-2018 P-rare

Viewpoint: GlaxoSmithKline abandonment of rare disease gene-therapy drug program because it’s not a money generator hurts children

When GlaxoSmithKline, long a global leader in the effort to pioneer gene replacement therapies, announced it would halt its drug ...
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Cryogenics logistics: Delivering CAR-T treatments at minus 240 degrees to save lives

Last year, the FDA approved the first CAR T-cell treatments—a new class of promising therapies that train the body’s immune ...
3-1-2018 Cancer-chemotherapy-001

Immunotherapy mystery: Drugs treat ovarian cancer ‘when they should not have’

[Four women with rare ovarian cancer], strangers to one another living in different countries, asked their doctors to try new ...
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Transforming modern medicine doesn’t have to be high tech or expensive

While intricate high-tech breakthroughs are incredible, it's important to realize the value of low-cost and low-tech ingenuity ...
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CAR-T cell therapy could drive a ‘revolution’ in cancer treatments

[T]he United States Food and Drug Administration (FDA) made the landmark decision to approve two ‘drugs’ that use CAR-T cell ...
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Esophageal cancer treated with risky modified T-cell therapy in China

Esophageal cancer is one of the most common forms of cancer in China. Like many other types, cancer of the esophagus ...
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Cutting gene therapy side effects by finding a better delivery system to the brain

Researchers have found a structure on the small viruses that deliver gene therapy that makes them better at crossing from ...
Stop AIDS? Gene therapies target 'almost impossible to cure' HIV

Stop AIDS? Gene therapies target ‘almost impossible to cure’ HIV

Experts say gene therapy could finally be used to treat HIV and AIDS - after a decade of trying to replicate the ...
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Video: Biohacker documents his own DIY gene therapy

DIY gene therapy is far from the mainstream, but the numbers of people who are attempting to genetically engineer their ...

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