(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.
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
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
UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called “Bubble Baby” disease, a life-threatening condi... Read Post