Medical research

Researchers develop 'dimmer switch' to help control gene therapy

In a major advancement in the field of gene therapy for rare and devastating diseases, researchers at Children's Hospital of Philadelphia (CHOP) have developed a "dimmer switch" system that can control levels of proteins ...

Diseases, Conditions, Syndromes

RNA modification may protect against liver disease

A chemical modification that occurs in some RNA molecules as they carry genetic instructions from DNA to cells' protein-making machinery may offer protection against non-alcoholic fatty liver, a condition that results from ...

Diseases, Conditions, Syndromes

Preliminary results of clinical trial for Crigler-Najjar syndrome

Preliminary results from the European gene therapy trial for Crigler-Najjar syndrome, conducted by Genethon in collaboration with European network CureCN, were presented at the EASL (European Association for the Study of ...

Genetics

Gene discovery may hold key to better therapies for OCD

In the first analysis of its kind, researchers at Columbia University Vagelos College of Physicians and Surgeons and several other institutions have linked distinct patterns of genetic mutations with obsessive-compulsive ...

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Gene therapy

Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods.

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