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Stem Cell Related Patent Number US5888983
| Title: | Method and oligonucleobase compounds for curing diseases caused by mutations | | Inventors: | Kmiec, Eric B.; Yardley, PA, USA
Cole-Strauss, Allyson D.; Narbeth, PA, USA | | Summary: | Described herein are double-stranded oligonucleotides containing a human wild-type allele. The invention relates to oligonucleotides comprised of a first region and a second region, such that each region has at least 15 nucleotides, each of which in the first region is Watson-Crick paired to a nucleotide of the second region to form at least one ribo-type segment of at least 3 nucleotides, and such that the sequence of the first or second region is the sequence of a fragment of a wild-type allele of a human gene. Further disclosed are methods of introducing specific alterations into the genome of cells that have been removed from a human subject suffering from a medical condition that is the result of a genetic lesion, such that the cells may include hematopoietic stem cells and hepatocytes, and such that the specific alteration in the genome is designed to correct the genetic lesion via the introduction of an oligonucleotide compound containing ribonucleotides and deoxyribonucleotides into the cells which are thereafter reintroducing into the subject, and such that the ribonucleotides of the compound may contain 2-substituents that enhance their resistance to Rnase. Claims of the invention include examples for the therapeutic use of such oligonucleobase compounds in the treatment of a wide variety of disease. | | Abstract: | The invention concerns methods of introducing specific alterations in the genome of cells that have been removed from a subject suffering from a medical condition that is the result of a genetic lesion. The specific alteration is designed to correct the genetic lesion. The method comprises introducing an oligonucleotide compound, containing ribonucleotides and deoxyribonucleotides, into the cells and thereafter reintroducing the cells into the subject. Specific types of cells include hematopoietic stem cells and hepatocytes. The ribonucleotides of the compound can have 2-substituents that enhance their resistance to RNase. | | US Patent Website: | Click Here for Full Text of Patent | | Title Number: | US5888983 | | Application Number: | US1997000906265 | | Date Filed: | 05/08/1997 | | Date Published: | 30/03/1999 | | Assignee: | Thomas Jefferson University, Philadelphia, PA, USA |
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