Table of Contents
What is Aavdj?
AAV-DJ is a highly recombinogenic hybrid vector created from DNA shuffling of eight AAV serotypes, which mediates efficient gene expression both in vitro and in vivo.
What is the difference between AAV and rAAV?
rAAV are more likely than wild-type AAV to integrate at non-homologous sites in the genome, and do so at about a 0.1% frequency. Nonetheless, the majority of rAAV particles are thought to be maintained in episomes or concatemers. Episomes differ profoundly from viral particles produced during a lytic cycle.
Is AAV a virus?
Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae.
How Does gene therapy work?
With gene therapy, doctors deliver a healthy copy of a gene to cells inside the body. This healthy gene may replace a damaged (mutated) gene, inactivate a mutated gene or introduce an entirely new gene. Carriers, called vectors, transport these healthy genes into cells.
What is rAAV gene therapy?
The use of recombinant adeno-associated viruses (rAAVs) ushered in a new millennium of gene transfer for therapeutic treatment of a number of conditions, including congenital blindness, hemophilia, and spinal muscular atrophy.
What is AAV and adenovirus?
Adenoviruses and Adeno-Associated Viruses (AAVs) are two types of viral vectors used for gene delivery. Both of these recombinant viral systems have the ability to infect a broad range of hosts, including dividing and non-dividing cells, without integrating with the host genome.
What is AAV based therapy?
AAV vectors are bioengineered tools that use a non-enveloped virus to transport modified genetic material safely into tissues and cells impacted by otherwise difficult-to-treat conditions.
What diseases can be cured with gene therapy?
Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.
Can AAV be used in vitro?
In recent years, adeno-associated viruses (AAVs) have been used for many in vitro and in vivo applications due to their high transduction efficiency, safety, and extended stable gene expression. Furthermore, several recent clinical trials demonstrated the full potential of AAVs for human gene therapy1,2,3,4.
Is AAV secreted?
AAV was found to be secreted by an unknown mechanism into cell culture media using adherent HEK293 cells and harvesting rAAV from the media at a late time-point post-transfection.
What is an rAAV?
Recombinant adeno-associated virus (rAAV) vectors are unique among the vector classes currently available for human gene therapy in that they are based upon a class of viruses that commonly inhabits a human host without causing any detectable pathology.
What does rAAV stand for?
Recombinant adeno-associated virus (rAAV) based genome engineering is a genome editing platform centered on the use of recombinant rAAV vectors that enables insertion, deletion or substitution of DNA sequences into the genomes of live mammalian cells.