|
|
|
Stem Cell Related Patent Number US6312956
| Title: | Nuclear targeted peptide nucleic acid oligomer | | Inventors: | Lane, Kirk B.; Brentwood, TN, USA | | Summary: | Described herein is a novel composition comprised of a nuclear localization sequence linked to a peptide nucleic acid by a single peptide bond. The invention relates to a composition composed of a nuclear localization sequence and a peptide nucleic acid oligomer (NLS-PNA), such that 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 uses emphasize gene therapy in the treatment of diseases that can be genetically targeted, such as heart disease, cancer, cerebrovascular diseases, cirrhosis, chronic pulmonary diseases, and human immunodeficiency virus. | | 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: | US6312956 | | Application Number: | US1999000411706 | | Date Filed: | 01/10/1999 | | Date Published: | 06/11/2001 | | Assignee: | Vanderbilt University, Nashville, TN, USA |
|
|
