摘要:
The present invention is directed to nucleic acid molecules containing a loop sequence designed to circumvent exportin-5 mediated export, and methods using these novel molecules.
摘要:
The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.
摘要:
The present invention provides novel pseudotyped retroviral vectors that can transduce human and other cells. Vectors are provided that are packaged efficiently in packaging cells and cell lines to generate high titer recombinant virus stocks expressing novel envelope glycoproteins. The present invention further relates to compositions for gene therapy.
摘要:
The present invention provides an adenovirus serotype 30 (Ad30) fiber amino acid sequence. The present invention also provides polynucleotides and expression vectors encoding an Ad30 fiber and viral particles and cells containing such expression vectors. The present invention further provides methods of treating genetic diseases or cancers in a mammal using the polynucleotides, polypeptides, expression vectors, viral particles and cells of the present invention.
摘要:
A controlled release delivery system includes a functional gene vector in a biodegradable polymeric microsphere encapsulating the vector. The present invention further provides a method of making a controlled release delivery system by encapsulating the functional gene vector in a biologically degradable polymeric microsphere.
摘要:
This invention provides recombinant adenoviral vectors having an expression control sequence operatively linked to a gene that encodes an anti-inflammatory polypeptide or that produces on transcription an anti-inflammatory ribozyme or antisense RNA molecule. This invention also provides methods of transferring a gene to a synovial cell by transducing the synovial cell in vivo, with a recombinant adenoviral vector. This invention also provides methods of treating an inflammatory condition in the joint of a subject comprising administering to the joint a therapeutically effective amount of a recombinant adenoviral vector having an expression control sequence operatively linked to a gene that encodes an anti-inflammatory polypeptide or that produces on transcription an anti-inflammatory ribozyme or antisense RNA molecule.
摘要:
The present invention is directed to RNA interference (RNAi) molecules targeted against a Huntington's disease nucleic acid sequence, and methods of using these RNAi molecules to treat Huntington's disease.
摘要:
The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence that encodes poly-glutamine repeat diseases, and methods of using these RNAi molecules.
摘要:
The present invention is directed to RNA interference (RNAi) molecules targeted against a nucleic acid sequence that encodes poly-glutamine repeat diseases, and methods of using these RNAi molecules.
摘要:
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.