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Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. [1] Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases. [ 2 ]
Recombinant adeno-associated virus (rAAV) based genome engineering is a genome editing platform centered on the use of recombinant AAV vectors that enables insertion, deletion or substitution of DNA sequences into the genomes of live mammalian cells.
Because of AAV's specialized gene therapy advantages, researchers have created an altered version of AAV termed self-complementary adeno-associated virus (scAAV). Whereas AAV packages a single strand of DNA and must wait for its second strand to be synthesized, scAAV packages two shorter strands that are complementary to each other.
One disadvantage is that they are not able to carry large amounts of foreign genetic materials. Furthermore, the need to express the complementary strand for its single-stranded genome may delay transgene expression. [45] As of 2020, 11 different AAV serotypes—differing by capsid structure and consequently by tropism—had been identified. [43]
One example is the so-called vision restoration gene therapy, that aims to restore vision in patients with end-stage retinal diseases. [ 70 ] [ 71 ] In end-stage retinal diseases, the photoreceptors, as the primary light sensitive cells of the retina are irreversibly lost.
Tripod Beta is an incident and accident analysis methodology made available by the Stichting Tripod Foundation [1] via the Energy Institute.The methodology is designed to help an accident investigator analyse the causes of an incident or accident in conjunction with conducting the investigation.
A lentiviral vector carrying therapeutic transgenes are then introduced to the culture to infect them. The now modified cells continue to be cultured until they can be infused into the patient. In vivo gene therapy is the sample injection of viral vectors containing transgenes into the patient. [5]
If the transgene is incorporated into somatic cells, the transgene will stay with the somatic cell line, and thus its host organism. [ 6 ] Gene delivery is a necessary step in gene therapy for the introduction or silencing of a gene to promote a therapeutic outcome in patients and also has applications in the genetic modification of crops.