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Stem Cell Related Patent Number US6623966
| Title: | Nuclear targeted peptide nucleic acid oligomer | | Inventors: | Lane, Kirk B.; Brentwood, TN, USA | | Summary: | This invention describes methods of increasing the expression of a wild-type nucleic acid and inhibiting transcription of a mutant nucleic acid with PNA oligomers and nuclear localization sequences. The invention relates to a composition comprising a nuclear localization sequence and a peptide nucleic acid oligomer (NLS-PNA), and the inhibited transcription of a mutant nucleic acid is described as having a mutation in a eukaryotic cell. Claims of the invention include uses of the composition in the regulation of gene expression, in gene therapy, and in the production of pharmaceutical nucleic acids and proteins. Also provided are methods by which the NLS-PNA may be applied to scientific and therapeutic transfection and the expression of nucleic acids in cells types that previously were resistant to transfection and therapy, such as quiescent cells, differentiated cells, embryonic stem cells, and eukaryotic cells with intact nuclear membranes. Further described are methods by which the NLS-PNA may be combined with a membrane transport sequence (MTS) forming a novel compound designated as an MTS-NLS-PNA wherein the MTS provides transport through the cytoplasmic membrane of a cell. Therapeutic applications of a nuclear targeted peptide nucleic acid oligomer include treatment for genetically based diseases, and for diseases that can be treated genetically including heart disease, cancer, cerebrovascular diseases, chronic pulmonary diseases, and human immunodeficiency virus infection. | | Abstract: | A composition comprising a nuclear localization sequence and a peptide nucleic acid oligomer (NLS-PNA) is described. Uses of the composition include, but are not limited to: regulation of gene expression, gene therapy, and the production of pharmaceutical nucleic acids and proteins. In addition, the NLS-PNA is useful for scientific and therapeutic transfection and expression of nucleic acids in cells types that previously were resistant to transfection and therapy including quiescent cells, differentiated cells, embryonic stem cells, and eukaryotic cells with intact nuclear membranes. The NLS-PNA can be combined with a membrane transport sequence (MTS) forming a novel compound referred to as an MTS-NLS-PNA wherein the MTS provides transport through the cytoplasmic membrane of a cell. A nuclear targeted peptide nucleic acid oligomer is useful for the treatment of genetic based diseases and diseases that can be treated genetically including heart disease, cancer, cerebrovascular diseases, chronic pulmonary diseases, human immunodeficiency virus, and other diseases, conditions and disorders. | | US Patent Website: | Click Here for Full Text of Patent | | Title Number: | US6623966 | | Application Number: | US2001000935032 | | Date Filed: | 22/08/2001 | | Date Published: | 23/09/2003 | | Assignee: | Vanderbilt University, Nashville, TN, USA |
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