Adeno-associated virus(AAV) is a small,non-enveloped virus that contains a single-stranded DNA genome. It was the first gene therapy drug approved in the Western world in November 2012 to treat patients with lipoprotein lipase deficiency. AAV made history and put human gene therapy in the fore...
Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene therapy owing to its minimal pathogenicity and ability to establish long-term gene expression in different tissues. Recombinant AAV (rAAV) has been engineered for enhanced specificity and developed as a tool for ...
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the growt...
Diseases of the central nervous system (CNS) have provided enormous opportunities for the therapeutic application of viral vector gene transfer. Adeno-associated virus (AAV) has been the vector of choice in recent clinical trials of neurological disease, including Parkinson's and Alzheimer's disease,...
Affinia Therapeutics is expanding what's possible in adeno-associated virus (AAV) gene therapy today and bringing transformative medicines for people.
Severaltrialswith AAV are on-going or in preparation, mainly trying to treat muscle and eye diseases; the two tissues where the virus seems particularly useful. However, clinical trials have also been initiated where AAVvectorsare used to deliver genes to the brain. This is possible because AAV...
(cell therapy), Adeno-Associated Viruses (AAV) have strengthened their position as a leading platform for gene delivery especially after recent landmark approvals: Luxturna (voretigene neparvovec), an AAV-based therapy that delivers a functional copy of the RPE65 gene to pat...
Directed evolution of adeno-associated virus (AAV) as vector for muscle gene therapy. Methods Mol. Biol. 2011, 709, 127-139. [PubMed]Yang L, Li J, Xiao X (2011) Directed evolution of adeno-associated virus (AAV) as vector for muscle gene therapy. Methods Mol Biol 709: 127 - 139...
Some of the unique features that make this virus attractive for gene therapy include the facts that AAV is prevalent in humans, it has never been identified as a causative agent of human disease, and it is able to insert its genome locus-specifically into human chromosomes. AAVs are members...
Corning Life Sciences walks through the gene therapy scale-up process, from small scale to large scale adeno-associated virus vector (AAV) production.