Adeno-Associated Virus Vector Mobilization, Risk Versus Reality
نویسندگان
چکیده
منابع مشابه
Adeno-associated virus serotypes: vector toolkit for human gene therapy.
Recombinant adeno-associated viral (AAV) vectors have rapidly advanced to the forefront of gene therapy in the past decade. The exponential progress of AAV-based vectors has been made possible by the isolation of several naturally occurring AAV serotypes and over 100 AAV variants from different animal species. These isolates are ideally suited to development into human gene therapy vectors due ...
متن کاملAdeno-associated virus biology.
Adeno-associated virus (AAV) was first discovered as a contaminant of adenovirus stocks in the 1960s. The development of recombinant AAV vectors (rAAV) was facilitated by early studies that generated infectious molecular clones, determined the sequence of the genome, and defined the genetic elements of the virus. The refinement of methods and protocols for the production and application of rAAV...
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Repeated bouts of ischemia in the heart lead to fibrosis and eventually to heart failure. Although certain genes, such as SOD or hemoxygenase and antisense to AT(1)R, ACE, and (beta(1)-AR can provide short-term protection of the heart from ischemia, there is no known mechanism for constantly responding to repeated incidences of ischemia. We hypothesized that a "vigilant vector," designed to be ...
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Gene delivery vectors based on adeno-associated virus (AAV) have potential utility in the treatment of many genetic disorders. Current AAV vector manufacturing methods employ helper viruses to deliver functions needed to produce replication-defective recombinant AAV (rAAV) vectors, and clearance of infectious helper virus from the drug substance is essential for ensuring the safety of rAAV-base...
متن کاملAdeno-associated virus type 2 as vector for human gene therapy: Characterization of virus-host interactions
Background. Recombinant adeno-associated virus (rAAV) has many advantages for gene therapeutic applications in comparison to other vector systems. One of the most promising features is the ability of wild-type (wt) AAV to integrate site-specifically into human chromosome 19. However, this feature is lost in rAAV vectors due to the removal of the Rep coding sequences. Methods. HeLa cells were tr...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2020
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2020.118