Adeno Associated Virus (AAV) for Cell and Gene Therapy
Type
Blog Post
Published
Nov. 7, 2017, 1:59 p.m.
...disease is caused by a mutation in a single gene, it can potentially be treated by correcting the mutation.... However, many diseases require techniques that can deliver gene therapies directly to the cells that... tools such as nanoparticles. These technologies can have serious adverse effects, and their immunogenic... human population. These pre-existing antibodies can interfere with virus-cell interactions and seriously...the immune system – patients’ own immune systems can develop antibodies that neutralize the AAV vectors...disease, and HIV. A notable landmark study showed significant clinical efficacy against a form of blindness... of LCA and mutations in RPE65 (n=3), showed significant improvements in vision over a period of 1 to ...