| abstract |
The present invention provides regulatory elements and vectors containing such regulatory elements that can be used to stimulate transcription of relevant genes in certain tissue types. The transcriptional regulatory elements described herein are operably linked to a transgene, such as acid alpha-glucosidase (GAA), to promote expression of the GAA transgene in cells, such as muscle cells, hepatocytes, or neurons. The transcriptional regulatory elements described herein are operably linked to a therapeutic transgene and can be used to treat a variety of disorders, such as lysosomal storage diseases, particularly Pompe disease. |