Adeno-Associated Virus


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De·pen·do·vi·rus

(dē-pen'dō-vī'rŭs),
A genus of small defective single-stranded DNA viruses in the family Parvoviridae that depend on adenoviruses for replication.
[L. dependeo, to be dependent upon, + virus]

Adeno-Associated Virus

A member of the parvovirus family, which is composed of small viruses with a genome of a single-stranded DNA. AAVs insert genomic material at a specific site on human chromosome 19 with nearly 100% certainty, and are used to construct vectors that introduce genes into cultured cells. There is virtually no downside to AAV vectors, given the small amount of DNA they can carry and because AAV is non-pathogenic (most people are AAV carriers). In contrast to adenoviruses, AAV usually does not trigger an immune response to cells infected with it, and thus can deliver genes to sites of interest, including the brain in the context of gene therapy for diseases of muscle and eye, tissues where AAV seems to be most useful.
References in periodicals archive ?
Xiao, "Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo" Gene Therapy, vol.
He and his associates have focused on the relatively nonpathogenic adeno-associated virus.
Protective effect of recombinant adeno-associated virus 2/8-mediated gene therapy from the maternal hyperphenylalaninemia in offsprings of a mouse model of phenylketonuria.
"There is a major difference between adeno-associated virus vectors and adenovirus vectors,' he says.
A member of this family, the avian adeno-associated virus (AAAV) has been fully characterized [3, 8] and successfully used for gene delivery in chicken embryo tissues and cells [7, 8].
In a phase II trial, researchers used an adeno-associated virus to deliver the cystic fibrosis transmembrane conductance regulator gene to the lung epithelium.
Phase I trial results using an adeno-associated virus that is injected into muscle to provide factor IX offer hope for patients with this disease.
Selecta Biosciences and Asklepios BioPharmaceutical announced a strategic partnership to jointly develop, manufacture and commercialize targeted therapeutics for life-changing, next-generation adeno-associated virus gene therapies in areas of high medical need.
This product is an adeno-associated virus vector-based gene therapy that targets the cause of SMA.
Currently, more than 130 companies in China are developing cell and gene therapies ranging from chimeric antigen receptor T cell therapy (CAR-T) / T cell receptor therapy (TCR-T) and adeno-associated virus (AAV) to oncolytic virus.
Pall Corporation and Cell and Gene Therapy Catapult, will work with Cobra Biologics to investigate continuous manufacturing of adeno-associated virus (AAV) for gene therapy applications.