Access to genetic testing brings with it several advantages – one being early diagnosis for young patients with potentially life-threatening multi-system conditions. Retinal-renal syndrome ...

For instance, candidate Huntington’s drugs that aim to reduce activity of the mutated gene (or the protein it codes for) have struggled in clinical trials. The new study implies that because very few ...

Scientists have found 697 genetic variants – or small changes within a gene – linked to depression, of which almost 300 were previously unknown. A Queensland scientist described the project as ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

Many animals survived even after undergoing radiation-based genetic mutations—notably, a number of pet dogs abandoned by their owners during Chernobyl’s hasty evacuation orders. Today ...

The company is submitting the Hunter syndrome treatment for U.S. approval on a rolling basis, a process it expects to complete this quarter. An approval would make RGX-121 the first gene therapy for ...

Abuhelwa Z, Ali NHA, Xie Z, et al. The argument for targeted therapy in IDH1 mutated myelodysplastic syndromes (MDS): poor outcomes post-hypomethylating agent failure in higher risk MDS and ...

The first human trial of insulin-producing cells that have been gene-edited to evade immune attack is a success so far. The cells have survived and produced insulin for a month after being ...

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Errors in the EGFR gene are one type of lung cancer biomarker. EGFR, which stands for epidermal growth factor receptor, is a protein located on the surface of cells that helps them grow. A mutation in ...

Scientists led by a team at Columbia University have trained a model to predict how the genes inside a cell will drive its behavior, which could be a powerful tool with the potential to broaden ...