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.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.
- What are recombinant adeno-associated viral vectors?
- What does adeno-associated virus do?
- What is the difference between adenovirus and adeno-associated virus?
- Why are recombinant adeno-associated viruses used in gene therapy?
- What is adeno-associated virus 9?
- Are Aavs safe?
- Does recombinant AAV cause disease in humans?
- Do adenoviruses integrate?
What are recombinant adeno-associated viral vectors?
Adeno-associated viral vectors (AAV) are based on the adeno-associated virus, a non-pathogenic, single-stranded DNA parvovirus. Recombinant AAV (rAAV) vectors' excellent safety profile and durable gene expression have made it the preferred vector for gene therapy in the nervous system.
What does adeno-associated virus do?
The viruses cause a very mild immune response. Several additional features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models….
| Adeno-associated virus | |
|---|---|
| Family: | Parvoviridae |
| Subfamily: | Parvovirinae |
| Genus: | Dependoparvovirus |
| Viruses included: |
What is the difference between adenovirus and adeno-associated virus?
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….Adenovirus vs. AAV.
| Adenovirus | AAV | |
|---|---|---|
| Protein Expression | High | Low |
| Gene Expression | Transient | Potentially Long Lasting |
| Target Cell's Immune Response | High | Very Low |
Why are recombinant adeno-associated viruses used in gene therapy?
Adeno-associated virus (AAV) vectors have been frequently used for gene therapy applications due to the lack of pathogenicity and toxicity, ability to infect both dividing and non-dividing cells, and long-term transgene expression profiles. Furthermore, the safety profile in various clinical trials has been good.
What is adeno-associated virus 9?
9. AAV is a small, helper-dependent parvovirus consisting of a single-stranded 4.7-kb DNA genome surrounded by a simple, naked icosahedral capsid. The AAV vector is attractive for use in gene therapy because it is efficient, long-lived, and non-toxic.
Are Aavs safe?
To date, AAV has been shown to be safe and effective in preclinical and clinical settings. AAV can be used in a wide range of clinical applications in multiple diseases due its unique biological and biophysical properties.
Does recombinant AAV cause disease in humans?
Though AAV is not known to cause disease in humans, precautions must be taken due to the possibility of insertional mutagenesis. In addition, helper viruses used to trigger AAV replication may cause disease. Numerous serotypes of adeno-associated virus with different cell tropisms have been isolated.
Do adenoviruses integrate?
Recent studies have shown that replication-incompetent adenoviral vectors randomly integrate into host chromosomes at frequencies of 0.001-1% of infected cells.