منابع مشابه
Versatility of AAV vectors for retinal gene transfer
Gene therapy represents a promising therapeutic option for many inherited and acquired retinal diseases. Recombinant adeno-associated viral vectors (AAV) are the most efficient tools to transfer genes in vivo to the retina. The recent identification of dozens of novel AAV serotypes enormously expands on the versatility of AAV as vector system for in vivo somatic gene transfer. The results from ...
متن کاملOcular gene transfer with self-complementary AAV vectors.
PURPOSE Self-complementary AAV (scAAV) vectors have been developed to circumvent rate-limiting second-strand synthesis in single-stranded AAV vector genomes and to facilitate robust transgene expression at a minimal dose. In this study, the authors investigated the effects of intraocular injections of type 2 scAAV.GFP in mice. METHODS Dose-response experiments were performed to compare conven...
متن کاملSyngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here we systemically evaluated the biological properties of three types of "empty" ...
متن کاملOptimized AAV rh.10 Vectors That Partially Evade Neutralizing Antibodies during Hepatic Gene Transfer
Of the 12 common serotypes used for gene delivery applications, Adeno-associated virus (AAV)rh.10 serotype has shown sustained hepatic transduction and has the lowest seropositivity in humans. We have evaluated if further modifications to AAVrh.10 at its phosphodegron like regions or predicted immunogenic epitopes could improve its hepatic gene transfer and immune evasion potential. Mutant AAVr...
متن کاملDiverse Efficiencies in Systemic Muscle Gene Transfer with Different Serotypes of Aav Vectors
INTRODUCTION: Delivery of gene vectors to local muscle or heart tissue has been achieved by direct intramuscular (i.m.) injection or by local blood vessel perfusion of viral vectors, particularly the AAV vectors. However, a major challenge is how to deliver the therapeutic genes into most, if not all, of the diseased muscles. Previous efforts to deliver genes into multiple muscles have relied o...
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ژورنال
عنوان ژورنال: Japanese Journal of Thrombosis and Hemostasis
سال: 2008
ISSN: 1880-8808,0915-7441
DOI: 10.2491/jjsth.19.265