Genetics

Promising gene replacement therapy moves forward

Research led by Dr. Krystof Bankiewicz, who recently joined The Ohio State University College of Medicine, shows that gene replacement therapy for Niemann-Pick type A disease is safe for use in nonhuman primates and has therapeutic ...

Genetics

Researchers redefine the footprint of viral vector gene therapy

Building on a track record of developing adeno-associated viral (AAV) vectors as a groundbreaking clinical tool for gene therapy and gene editing, Children's Hospital of Philadelphia (CHOP) researchers report a more sensitive ...

Neuroscience

Economic, fast and efficient cell-type targeting

It is a remarkable proof for the concept of IOB: working closely hand in hand, our molecular and clinical researchers have developed a library of 230 adeno associated viral vectors (AAVs), each with a different synthetic ...

Neuroscience

Retina restructures itself after cell death

Following gene therapy, the retina can restructure itself and regain normal light responses, according to research in mice published in JNeurosci. These results emphasize the plasticity of the retina and support ongoing development ...

page 1 from 23

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