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Genes ex silico: Computer-designed virus yields phenotype expression benefits

(PhysOrg.com) -- Gene therapy is medicine?s rising star with adeno-associated virus (AAV) vectors ? nonpathogenic parvoviruses ? among the most promising supporting actors, due largely to their capability to integrate into transcriptionally silent genomic regions (areas that do not, via RNA polymerases, make a messenger RNA copy of DNA-stored genetic information). That being said, there?s been a downside in assembling AAV vectors into adenoviral (Ad) viral backbones, which are used extensively in genetic research and therapy: They rely on replication (Rep) proteins ? in this study, the Rep 78/68 polypeptide ? which limit viral amplification methodologies.
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News From The Journal Of Clinical Investigation, July 1, 2009

Health : Medical News Today (5 years ago)

GENE THERAPY: Defining immune pathways limiting gene therapy In gene therapy, recombinant adeno-associated viruses (AAVs) are commonly used vehicles for delivering the therapeutic gene into target cells. One factor limiting the clin... Read Post

Success Of Gene Therapy Will Depend On Ability To Advance Viral Delivery Vectors To Commercialization, As Described In Human Gene Therapy

Health : Medical News Today (3 years ago)

Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues. One technology platform that ... Read Post

Liver Gene Therapy Corrects Heart Symptoms in Model of Rare Enzyme Disorder

Health : Newswise Medical News (yesterday)

Researchers examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with a rare enzyme deficiency disorder. The works describes how an injection of a vector expressing the IDUA enzyme to the... Read Post


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