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Disruptive Science

Meet the Team: Homegrown Therapies Get Ready to Fight NASH

Meet the Team: Homegrown Therapies Get Ready to Fight NASH

Meet the Team: Homegrown Therapies Get Ready to...

One of the biggest public health problems of our time is likely soon to face off with a new wave of cutting-edge medicines. As non-alcoholic steatohepatitis (NASH), a severe form of non-alcoholic fatty liver disease, or NAFLD, continues to be a growing public health threat, scientists are racing...

How Next-Generation Sequencing Is Helping to Build Better Vaccines

How Next-Generation Sequencing Is Helping to Build Better Vaccines

How Next-Generation Sequencing Is Helping to...

If you’ve ever tried an at-home genetic testing kit, you’re familiar with whole-genome sequencing, the technology that allows people to quickly and easily map their DNA. This same technology is now being used to track the genomes of potentially deadly microbes. ...

Capturing the Itch: Using Digital Wearable Devices to Help Patients with Atopic Dermatitis

Capturing the Itch: Using Digital Wearable Devices to Help Patients with Atopic Dermatitis

Capturing the Itch: Using Digital Wearable...

For people who have atopic dermatitis (AD) — an autoimmune condition that causes painful, itchy, and inflamed skin — symptoms often flare up at night, leading to disrupted sleep. Until recently, there hasn’t been a way to truly capture how much a person’s quality of life is impacted by these...

Designing Medicines to Go Where They're Needed: Lessons from Liver Targeting

Designing Medicines to Go Where They're Needed: Lessons from Liver Targeting

Designing Medicines to Go Where They're Needed:...

Designing transformational medicines for patients requires them to be efficacious and safe.  Sometimes, drug molecules have the desired effect in the organ of treatment and undesired effects in other organs, meaning one way to achieve a safer medicine is through directing the drug molecule to the...

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...

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. ...

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