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Foundations of Science

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...

The Evolution of Inflammation

The Evolution of Inflammation

The Evolution of Inflammation

Hunting wooly mammoths with stone age technology took guts. If the archaeological record is to be believed, it also took an incredible ability to bounce back from serious injuries. ...

What is Green Chemistry?

What is Green Chemistry?

What is Green Chemistry?

The way contemporary chemists work is a lot different than the way their predecessors worked only a few generations ago. You might think that the change is due to new technologies or breakthroughs in scientific understanding, but that’s only partly right. One of the major reasons chemistry is...

The Good, the Bad and the Strange of Physical Pain

The Good, the Bad and the Strange of Physical Pain

The Good, the Bad and the Strange of Physical...

Most people struggle with pain at some point in their life, and when it gets bad enough it can be a debilitating condition. However, while pain has its obvious and sometimes devastating downside, our ability to feel physical pain is also part of maintaining our health. For a time, physicians even...

Inside the Toxicology Lab: From Guardians of the Genome to Bottleneck Breakers and Beyond

Inside the Toxicology Lab: From Guardians of the Genome to Bottleneck Breakers and Beyond

Inside the Toxicology Lab: From Guardians of...

When it comes to creating new medicines, the stories that most often reach the public are the discoveries of new biological pathways or novel compounds. But in the nearly decade-long journey a medicine takes to get from the lab to patients, drug safety studies are an integral, though often behind...

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