Advancing Medical Research
Foundations of Science
Using "Molecular Glues" to Hijack the Body’s Garbage Disposal System
Proteins are the stuff of life. They comprise the hormones, enzymes and many other molecules that are essential to our body’s functioning. But sometimes these loyal workhorses can go rogue, leading to cancer, autoimmune diseases and a variety of neurological conditions.
From Flask to Vat: The Challenges of Scaling Up Gene Therapy
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.
But now, as these …
Hope in Translation: Bringing Gene Therapy to the Clinic
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 dystrophin gene sparked hope that scientists could someday replace the mutated gene with a…
Moving Beyond Clinical Trials: 4 Lessons From Real-World Data
Clinical trials continue to be the gold standard for studying the safety and efficacy of a new medicine. They are designed to take place in very controlled environments, in a well-defined population, to understand the effects of a medicine on the specific patients; however, these experimental conditions don’t always…
Cytokines of Interest: Understanding Their Paths to Disease
In the diverse universe of cytokines, signaling proteins secreted by our cells that affect nearly every biological process in the body, there is a specific subset that scientists pay extra attention to for their role in…
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.
Gene therapy uses…
Touching Base: the Speed and Accessibility of Next Generation Sequencing
When German engineer Carl Benz took the world’s first automobile out for a spin in 1886, most observers were underwhelmed. With an engine that generated 0.75 horsepower and a top speed of 10 miles per hour, not many people rushed out to buy Benz’s first car. The $1000 price tag, an outrageous sum at the time, also discouraged all but the wealthiest would-be auto enthusiasts.
But subsequent generations of…
Bespoke Is Often Better: How Scientists Are Customizing Gene Therapy
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.
But in recent years, this tale has become more complex. Scientists are increasingly aware that a…