Three 2026 Breakthrough Prize winners reflect on developing Luxturna, a gene therapy that treats blindness caused by rare ...

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 ...

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 ...

A gene therapy pioneer whose invention was pivotal to a first-of-its-kind personalized gene-editing treatment for a Philadelphia-area infant is among the winners of this year’s Franklin Institute ...

Stuart Orkin and Swee Lay Thein shared a Breakthrough Prize in Life Sciences for their research on genetic causes of sickle ...

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 ...

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 ...

A new prime assembly technique inserts DNA segments up to 11,000 base pairs into the genome, enabling correction of thousands ...

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 ...

Cell and gene therapy developers must build continuity of engagement to create trust in the modalities, according to a report ...

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 ...