Abstract:
The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.
Abstract:
The present invention is directed to viral vectors encoding small interfering RNA molecules (siRNA) targeted against a gene of interest, and methods of using these viral vectors.
Abstract:
The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.
Abstract:
The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.
Abstract:
The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.
Abstract:
The present invention is directed to viral vectors encoding small interfering RNA molecules (siRNA) targeted against a gene of interest, and methods of using these viral vectors.
Abstract:
The present invention is directed to viral vectors encoding small interfering RNA molecules (siRNA) targeted against a gene of interest, and methods of using these viral vectors.
Abstract:
The present invention provides polynucleotides and expression vectors containing a sequence encoding a soluble lysosomal enzyme and a sequence encoding Tat protein transduction domain (PTD), and the corresponding polypeptides. The present demonstrates the utility of these protein fusions in altering the bioavailability of proteins for use in treating genetic diseases or acquired diseases. The invention further provides cell expression systems, and methods of treating a genetic disease or cancer in a mammal using the polynucleotides, polypeptides, or expression system of the present invention.
Abstract:
The present invention is directed to viral vectors encoding small interfering RNA molecules (siRNA) targeted against a gene of interest, and methods of using these viral vectors.
Abstract:
The present invention is directed to small interfering RNA molecules (siRNA) targeted against nucleic acid sequence that encodes huntingtin or ataxin-1, and methods of using these siRNA molecules.