Medical research

A gene mutation that protects against disease

Canadian scientists looking at a rare genetic mutation think they may have discovered the proverbial fountain of youth. But it's not for everyone: so far, the mutation has only been found in a handful of French-Canadian families.

Genetics

Gene therapy startup to increase AAV gene therapy efficiency

Maximizing the efficiency of the adeno-associated virus (AAV) platform for gene therapy is the aim of a new pilot project of the National Institutes of Health (NIH). The NIH Platform Vector Gene Therapy (PaVe-GT) project ...

Genetics

Risk of AAV mobilization in gene therapy

New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy. These findings, which emphasize the need for mobilization resistant AAV vectors, are ...

Neuroscience

Neuroscientists advance understanding of pain from light touch

Researchers from the Pittsburgh Center for Pain Research at the University of Pittsburgh School of Medicine announced today in the journal Neuron that they've uncovered additional complexities behind mechanical allodynia—the ...

Genetics

Q&A: Getting to the roots of fragile X syndrome

The cause of fragile X syndrome (FXS), the most common inherited intellectual disability, is easy to see in the lab. Under electron microscopy, an affected X chromosome exhibits a deformed tip that gives the disorder its ...

page 1 from 40

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.

This text uses material from Wikipedia, licensed under CC BY-SA