Self-complementary AAV Vectors; Advances and Applications
نویسندگان
چکیده
منابع مشابه
Ocular 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 کاملDesign and construction of functional AAV vectors.
Using the basic principles of molecular biology and laboratory techniques presented in this chapter, researchers should be able to create a wide variety of AAV vectors for both clinical and basic research applications. Basic vector design concepts are covered for both protein coding gene expression and small non-coding RNA gene expression cassettes. AAV plasmid vector backbones (available via A...
متن کاملSleeping Beauty transposon vectors for therapeutic applications: advances and challenges
Transposable elements are natural, non-viral gene delivery vehicles capable of mediating stable genomic integration. The Sleeping Beauty (SB) transposon has the ability to cut-and-paste the ‘gene of interest’ into the genome, providing the basis for long-term, permanent transgene expression in transgenic cells and organisms. The SB transposon system is relatively well characterized, and has bee...
متن کاملAAV Vectors Vaccines Against Infectious Diseases
Since their discovery as a tool for gene transfer, vectors derived from the adeno-associated virus (AAV) have been used for gene therapy applications and attracted scientist to this field for their exceptional properties of efficiency of in vivo gene transfer and the level and duration of transgene expression. For many years, AAVs have been considered as low immunogenic vectors due to their abi...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2008
ISSN: 1525-0016
DOI: 10.1038/mt.2008.171