Adeno-Associated Virus(AAV) Gene Therapy Landscape Adeno-associated viruses (AAV) are virus particles composed of single-stranded DNA surrounded by a protein shell. Despite their simple structure, recombinant AAVs (rAAV) can perform the important role of delivering nucleic acids into cells during gene therapy. Due to the high demand for potentially curative treatments in areas of huge unmet […]
AAV protocol Download Adeno Associated Virus (AAV) a) Introduction of rAAV vector system Though wild-type AAV is not associated with human disease, it is naturally defective and requiring helper adenovirus or herpes simplex virus (HSV) coinfection for AAV replication, so recombinant AAV (rAAV) has been an attractive vector for gene therapy. In 1984, rAAV was […]
AAV protocol Download Adeno Associated Virus (AAV) Over the past decades, numerous AAV serotypes have been identified with variable tropism. To date, 12 AAV serotypes and over 100 AAV variants have been isolated from adenovirus stocks or human/nonhuman primate tissues. Among them, AAV2, AAV3, AAV5, AAV6 were discovered in human cells, while AAV1, AAV4, AAV7, […]
AAV protocol Download Adeno Associated Virus (AAV) AAV transduces cells through several stages: (1) viral binding to cell surface receptor/coreceptor, (2) endocytosis of the virus, (3) intracellular trafficking of the virus through the endosomal compartment, (4) endosomal escape of the virus, (5) intracellular trafficking of the virus to the nucleus and nuclear import, (6) virion […]
AAV protocol Download Adeno Associated Virus (AAV) The AAV shell is approximately 25nm in diameter and encapsidates a single-stranded DNA genome of 4.7 kilobases. The genome consists of two large open reading frames (ORFs) flanked by 145bp inverted terminal repeats (ITR), which are the only cis-acting elements required for AAV genome replication and AAV packaging. […]
AAV protocol Download Adeno Associated Virus (AAV) a) Advantages of AAV-mediated gene transferAAV has been developed into a very attractive candidate for creating viral vectors for gene therapy and the creation of isogenic human disease models due to various advantages. 1) Superior biosafety rating. The wild type AAV has not currently been known to cause […]
AAV protocol Download Adeno Associated Virus (AAV) AAV has been proved as the most excellent gene therapy vector. To date, more than 204 clinical trials have been carried out using AAV vectors for gene delivery [4], and promising gene therapy outcomes have been achieved from Phase 1, Phase 2 and Phase 3 trials for a […]
AAV protocol Download Adeno Associated Virus (AAV) Adeno-associated virus (AAV) is a small-single strand DNA virus, member of human parvovirus, originally described in the 1960s by Bob Atchison at Pittsburgh and Wallace Rowe at NIH as a contaminant in preparation of adenovirus [1,2]. Shortly after its discovery, AAV was then isolated from humans, but serological studies […]
AAV protocol Download Adeno Associated Virus (AAV) For normal tissues or organs, such as heart, liver, kidney, breast, pancreas, ovary, brain, eye, skeleton muscle, adipose tissue, etc., Genemedi systematically organizes the corresponding optimal AAV serotype, gene delivery method and injection volume for mouse and rat tissue infection. Recommended AAV gene delivery methods for different organs is […]
AAV protocol Download AAV capsids evolution and tissue-specific AAV discovery & development service The Genemedi help scientists and researchers in gene therapy vector development. Besides different scales of AAV production, we can also offer novel AAV discovery services with AAV re-engineering capsids library and sub-library generation based on different strategies, customized AAV capsids screening and […]
