Global Antisense and RNAi therapeutics Market is valued approximately at USD 3.69 billion in 2022 and is anticipated to grow with a healthy growth rate of more than 18.72% over the forecast period ...

Five families with children who have the newly discovered rare condition recently gathered in a Washington, D.C., park.

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) being an oral daily medication and nusinersen (Spinraza) being administered ...

BARCELONA, Spain, June 11, 2025 /PRNewswire/ -- SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today ...

The mRNA metabolism passively shapes the levels of an mRNA modification called m6A within a steady-state cell and upon stress.

Genetic vectors are vehicles for delivering foreign DNA into recipient cells. Vectors can replicate autonomously and typically include features to facilitate the manipulation of DNA as well as a ...

SB-007 is an investigational Protein Splicing dual AAV gene therapy in development for the treatment of Stargardt disease. It is designed to restore expression of the native full-length ABCA4 ...

BARCELONA, Spain, June 11, 2025 /PRNewswire/ -- SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today ...

Citrate is essential for the metabolism and development of neurons. A membrane transport protein called SLC13A5 plays a central role in this process and has previously been linked to a particularly ...

Scientists identified 473 human genes that act as genetic “on/off switches,” shaping disease risk through tissue-specific or ...