The gene mutation was isolated in a family in northern Italy where under half of the relations developed the disease. View on ...

New data from a Phase 1b clinical trial testing Fulcrum's pociredir for sickle cell disease are expected by the middle and ...

Orna Therapeutics and Vertex will use lipid nanoparticle system to develop gene-editing therapies for people with SCD and TDT ...

If a mutation occurs in proteins that regulate the growth of a cell ... These include cystic fibrosis and sickle cell anemia, to name a couple. Key Concepts in the Genetic Theory of Aging The key ...

Overall, the life expectancy of patients with SCD is lower than that of persons without SCD. 1 However, life expectancy varies depending on the type of disease, treatments administered, and ...

But these results also suggest that RAS mutations show a different biological behavior depending on the cell type where they are expressed, adding complexity to our understanding of RAS biology. RAS ...

This year saw several advancements across medicine, space technology, and AI that extend our knowledge in consequential ways.

Living with Sickle Cell Disease means that every treatment ... damaging cellular components such as lipids, proteins, and DNA. Over time, this can result in permanent organ damage and impaired ...

It is now 12 months since the Food and Drug Administration rolled out the historic approval of a CRISPR therapy designed for patients suffering from sickle cell anemia and the blood disorder beta ...