摘要:
The instant invention provides methods of expressing polynucleotides in the cells of the liver comprising administering viral particles comprising a recombinant AAV vector into a mammal, preferably a human.
摘要:
The invention pertains to a safe, quick and reliable method of detecting the presence of a target DNA sequence in a sample. The invention also pertains to a system for detecting presence of a target DNA sequence in a biological sample. The system includes an ITC template that includes a probe binding sequence that binds to a corresponding ITC probe and a first and second flanking primer recognizing sequence that binds to a first and second primer, respectively. The system also includes an ITC probe that binds to the ITC template probe binding sequence. The ITC probe includes a first marker molecule. The system also includes a first primer that binds to the first flanking primer sequence and a second primer that binds to said second primer sequence. The system further includes a target DNA sequence probe, wherein the target DNA sequence probe comprises a second marker molecule.
摘要:
Stocks of infectious rAAV are generated using yeast strains, bacterial strains, and bacteriophages engineered to express the required AAV proteins and harboring rAAV vector sequences. Stocks of rAAV virions of all serotypes and pseudotypes can be generated in prokaryotic and eukaryotic cells using the methods described herein.
摘要:
Vectors that encode Adeno-Associated Virus (AAV) Rep and Cap proteins of different serotypes and Adenovirus transcription products that provide helper functions were used to produce pseudotyped recombinant AAV (rAAV) virions. Purification methods generated pseudotyped rAAV virion stocks that were 99% pure with titers of 1×1012–1×1013 vector genomes/ml.
摘要:
The instant invention provides methods and materials for expressing a polypeptide with factor VIII activity comprising administering an rAAV vector encoding a truncated version of human factor VIII, containing, for example, a 90 kD heavy chain of factor VIII fused to a light chain of factor VIII.
摘要:
Suppressor tRNA's are used to regulate expression of transgenes that are toxic, or the expression thereof requires a factor that is toxic, to the host cell.