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References in periodicals archive ?
In gene therapy, the recombinant adeno-associated virus (rAAV) delivers DNA to target cells with reliable safety and high efficiency in preclinical and clinical settings.
- Adeno-associated virus (AAV) vector-based gene therapy became a principal actor in the development of therapies for monogenic diseases
Caption: Illustration of an adeno-associated virus used in gene therapy research.
The researchers used adeno-associated virus (AAV) to perform the gene therapy, which they said should help advance their work to human clinical trials quicker.
Researchers sent gene-editing components to cells in mice using the adeno-associated virus 9, and the rodents started producing dystrophin and showing improvement to skeletal muscle and the heart.
Methods: TB-4 recombinant adeno-associated virus (AAV) was constructed and induced to human NP cells.
Lysogene, based in Paris, has entered into a strategic collaboration with UMass Medical School and Auburn University of Auburn, Alabama, to develop preclinical studies using an adeno-associated virus (AAV) on GM1-gangliosidosis, "a rare, inherited disorder characterized by severe neurological impairment,'' according to a press release from Lysogene.
During the experiments, researchers, with the help of a genetically engineered adeno-associated virus that was injected into the retina, created a corrective copy of the gene and replaced it with the defective one.
Heth et al., "Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system," Proceedings of the National Academy of Sciences of the United States of America, vol.
Mandel used adeno-associated virus serotype 9 (V9), which has the unusual and attractive ability to cross the blood-brain barrier.
The gene can be delivered to the eye using a virus called Adeno-associated virus. The goal of the project is to begin testing the therapy in patients within the next five years.