Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of α-1-Antitrypsin Using Invasive and Noninvasive Delivery.

Recombinant AAV Serotype and Capsid Mutant Comparison for Pulmonary Gene Transfer of alpha-1-Antitrypsin Using Invasive and Noninvasive Delivery

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Résumé: Recent progresses in the characterization of physical properties of viruses allows for the first time to investigate distinct virus cycle events at the single particle level [1]. Using an original combination of experimental approaches based on Atomic Force Microscopy (AFM) and statistical physics modelization, we propose to study the physical properties of Adeno-Associated Virus (AAV) ...

Multilineage transduction of resident lung cells in vivo by AAV2/8 for α1-antitrypsin gene therapy

In vivo gene delivery has long represented an appealing potential treatment approach for monogenic diseases such as α1-antitrypsin deficiency (AATD) but has proven challenging to achieve in practice. Alternate pseudotyping of recombinant adeno-associated virus (AAV) vectors is producing vectors with increasingly heterogeneous tropic specificity, giving researchers the ability to target numerous...

Targeted Gene Delivery to the Enteric Nervous System Using AAV: A Comparison Across Serotypes and Capsid Mutants

Recombinant adeno-associated virus (AAV) vectors are one of the most widely used gene transfer systems in research and clinical trials. AAV can transduce a wide range of biological tissues, however to date, there has been no investigation on targeted AAV transduction of the enteric nervous system (ENS). Here, we examined the efficiency, tropism, spread, and immunogenicity of AAV transduction in...

Cloning and characterization of a bovine adeno-associated virus.

To better understand the relationship between primate adeno-associated viruses (AAVs) and those of other mammals, we have cloned and sequenced the genome of an AAV found as a contaminant in two isolates of bovine adenovirus that was reported to be serologically distinct from primate AAVs. The bovine AAV (BAAV) genome has 4,693 bp, and its organization is similar to that of other AAV isolates. T...