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Disruptive Science

Breaking the Rules of Biology to Beat Rare Genetic Diseases

Breaking the Rules of Biology to Beat Rare Genetic Diseases

Breaking the Rules of Biology to Beat Rare...

Like a skipping record, our genetic code has thousands of regions known as microsatellites where short combinations of base pairs — adenine (A), cytosine (C), guanine (G), and thymine (T) — repeat. For the most part, these repetitions are harmless. But in recent years, scientists have made...

Studying a Cell’s 'Messages in a Bottle' to Unlock Clues to Disease

Studying a Cell’s 'Messages in a Bottle' to Unlock Clues to Disease

Studying a Cell’s 'Messages in a Bottle' to...

Cells have a variety of ways of communicating with one another, cytokines and neurotransmitters being two such methods. ...

From Flask to Vat: The Challenges of Scaling Up Gene Therapy

From Flask to Vat: The Challenges of Scaling Up Gene Therapy

From Flask to Vat: The Challenges of Scaling Up...

Over the past two decades, gene therapy has been incubating at a very small scale in academic research labs. Like a chef fine-tuning a new recipe, scientists have been working in single batches that would only be sufficient to treat one or two patients at a time....

Hope in Translation: Bringing Gene Therapy to the Clinic

Hope in Translation: Bringing Gene Therapy to the Clinic

Hope in Translation: Bringing Gene Therapy to...

In 1987, scientists first uncovered the gene that is the target of mutations that causes Duchenne muscular dystrophy (DMD), a lethal muscle-wasting disease that affects about one in every 5,000 newborn boys. While the discovery of...

Inside

Inside "Custom Cars” For Gene Delivery

Inside "Custom Cars” For Gene Delivery

Gene therapy shows great promise to be potentially transformational treatments for a number of single-gene disorders, such as Duchenne muscular dystrophy, hemophilia and cystic fibrosis. ...

Bespoke Is Often Better: How Scientists Are Customizing Gene Therapy

Bespoke Is Often Better: How Scientists Are Customizing Gene Therapy

Bespoke Is Often Better: How Scientists Are...

When it comes to products from suits to kitchen cabinetry, bespoke often means better. That’s because custom-designed goods are more likely to fit your specific needs. ...

Breathing New Life Into Lung Cancer Research

Breathing New Life Into Lung Cancer Research

Breathing New Life Into Lung Cancer Research

The story of cancer was long seen as a single narrative: a cell acquires genetic mutations, starts to divide uncontrollably and then spreads. ...

New RNA Technology Could Get the Flu Vaccine Right, Every Year

New RNA Technology Could Get the Flu Vaccine Right, Every Year

New RNA Technology Could Get the Flu Vaccine...

We’ve been making the flu vaccine in nearly the same way for 70 years. A new technology based on RNA could disrupt that. ...

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