Rare diseases, defined by FDA as “a disease or condition that affects less than 200,000 people in the United States” (1), may ...

In the microscopic world of bacteria, gene transfer is a powerful mechanism that can alter cellular function, drive antibiotic resistance and even shape entire ecosystems. Now an interdisciplinary ...

In a breakthrough study, scientists have transferred a courtship behavior from one species to another, triggering the recipient to perform this completely foreign act as if it was its own. While genes ...

A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can ...

A 3D illustration of a transfer RNA molecule hovering over a messenger RNA. Transfer RNA acts as an adaptor to convert messenger RNA codons into amino acids during protein synthesis. Credit: ...

The inaugural Genetic Agency Technology Conference (GATC) on November 11th, 2025 will convene technical thought leaders, gene therapy developers, patients, their families, and patient advocates across ...

Significant unmet needs in SMA management include therapy access, fatigue, reproductive health, and emerging phenotypes in older patients. Intrathecal gene therapy offers potential for older SMA ...