The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

When 18 individuals with overlapping symptoms were left without a unifying diagnosis, Yale’s Saquib Lakhani and Lauren Jeffries joined forces with researchers around the world to discern the genetic ...

Lysosomal Storage Disorders are genetic conditions caused by enzyme deficiencies that lead to harmful substance buildup in cells, resulting in progressive organ damage. Common disorders in India ...

Genetic medicine has made huge progress over the past two decades. Gene therapy, gene editing, and mRNA treatments are now approved for a growing number of conditions. These approaches focus on fixing ...

Research on Tourette’s indicates that the condition is strongly influenced by inherited genetic factors, with environmental factors also contributing to its development. Tourette syndrome is a ...

Although mental disorders have multifactorial causes, genetics can explain some of them, yet this field remains largely unexplored in terms of guiding diagnoses and treatments. In the largest study of ...

A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...

Hunter Syndrome is a rare genetic condition that can cause damage to the body and the brain. It often causes children to pass away in their teens, but a UNC Health ...