A cutting-edge base-edited CAR T-cell therapy is opening a revolutionary new frontier for patients battling aggressive T-cell ...

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 ...

Researchers created a highly efficient gene-editing method that fixes multiple DNA mutations in a single step. The breakthrough could revolutionize genetic medicine by making treatments for complex ...

Patients with a previously untreatable form of blood cancer are now living disease-free after a world-first gene therapy. Alyssa Tapley, 16, became the first patient with an aggressive form of ...

In a recent study published in Nature Communications, researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications. Study: CRISPR-Cas9 ...

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 ...

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 ...

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 ...

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 ...

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 ...

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

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 ...