Adeno-associated virus inverted terminal sequences with repetition as promoter for in vivo gene transfer functional CFTR.

摘要

Describes the recombinant DNA constructs comprising the sequences DNA of the virus (AAV) COMMONWEALTH adeno MODIFIED ABLE TO OPERATE AS A eukaryotic expression vector for expressing DNA SEQUENCES STRANGE USING A NEW PROMOTER TRANSCRIPTION comprising END OF DNA AAV . It shows that the expression of a gene REPORTER TEST MAY BE OBTAINED FROM YOUR TABLE IN CELLS OF MAMMALS. ALSO it IS SHOWN THAT THIS COMBINATION OF VECTOR AND DEVELOPER MAY BE USED FOR ENTERING AND EXPRESSING A HUMAN GENE AND GENETIC DEFECT CORRECTING IN HUMAN CELLS RESULTING FROM THE MALFUNCTION OF A MUTANT GENE Endogenous. IN ADDITION, THE TABLE CAN BE USED TO GENETIC DEFECT CORRECTING to express a modified version of the human gene comprising a FUSION OF PORTION OF SAID GENE AND SYNTHETIC SEQUENCE CONTAINED ON THE TABLE.