Part A of LIGHTHOUSE Trial reinforce ATSN-201's favorable safety profile and durable structural and functional benefits in patients with X-linked retinoschisis ...

Comprehending the spate of recent rejections in the cell and gene therapy space may require looking no further than ...

Gene expression is controlled, in part, by the interactions between genes and regulatory elements located along the genome.

Ming, Professor of the Department of Chemical and Biological Engineering (CBE) at The Hong Kong University of Science and ...

OGM Can Resolve Previously Unresolved Rare Disease CasesOGM Can Serve as a Gold Standard Technique for Characterization of ...

Short QT syndrome is a genetic disease that leads to sudden cardiac death at a young age. Mutations in the SLC4A3 gene, which ...

Study in adults and children with aplastic anemia identifies parallel evolutionary pathways used by hematopoietic stem cells ...

A new genomic study reveals that butterflies and moths, despite diverging over 100 million years ago, use the same two genes—ivory and optix—to produce bright warning colors. The research shows that ...

A study reveals chromatin moves in constrained or freer patterns, shaping how genes contact regulatory elements and repair ...

National Eye Institute (NEI) scientists have found that the way the retina metabolizes glucose directly controls which genes get switched on and off in light-sensing photoreceptors. The findings ...

A bird swoops toward a bright orange-and-black moth resting on a leaf in the Amazon. It pulls up at the last second. The ...

Six-month data from adult Cohort 4 show structural and functional improvements and a favorable safety profile; early safety data from pediatric ...