New gene editing techniques, like adenine base editing and prime editing, are creating new possibilities for treating dilated cardiomyopathy, a heart condition affecting about 1 in 250 people ...

Encoded in our DNA are thousands of genes. Working together, they determine all sorts of physical attributes—from eye color and blood type to the number of limbs we have—and much, much more. It’s also ...

Over the past few decades, there has been remarkable progress in genetic manipulation technologies, bringing us closer to the point where genes can be modified in vivo. Such tools would open up the ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often too large to fit inside the viral vector used for delivery. Researchers at ...

Decades ago, researchers showed that Down syndrome (also known as trisomy 21), was caused when individuals carried an extra copy of chromosome 21. Down syndrome is also the most common genetic cause ...

This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged ...

For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that ...

Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where they perform their gene editing function. In this approach, a nanoparticle exposes cells to the ...

T therapy has helped previously untreatable for T-cell acute lymphoblastic leukemia patients achieve remission, with 82% responding and many now disease-free.

The human genome contains about 23,000 genes, but only a fraction of those genes are turned on inside a cell at any given time. The complex network of regulatory elements that controls gene expression ...

In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several measurement techniques commonly used in gene therapy. The study determined ...

"New technique reveals how gene transcription is coordinated in cells." ScienceDaily. www.sciencedaily.com / releases / 2024 / 06 / 240605162356.htm (accessed October 19, 2025).