| 摘要 |
The present invention provides new methods of administering recombinant AAV vectors for effective, long-term expression of a gene of interest in animals, including humans. Provided herein are methods of subcutaneously injecting recombinant AAV vectors containing a gene of interest encoding any diffusible polypeptide, ribozyme, nucleic acid, or antisense oligonucleotide for gene therapy. The methods of this invention provide significant advantages, including the ability to deliver any diffusible polypeptide or nucleic acid of a defined size via subcutaneous injection and obtain high-level, long-term expression in regions approximate or distant from the site of subcutaneous injection. |
