Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai
Patent
1997-08-05
1999-03-30
Elliott, George C.
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Carbohydrate doai
435 6, 4351723, 435325, 435366, 435372, 536 231, A61K 4800, C12Q 168, C12N 1511
Patent
active
058889833
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.
REFERENCES:
patent: 5760012 (1998-06-01), Kmiec et al.
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Yoon K. et al., "Targeted Gene Correction of Episomal DNA in Mammalian Cells Mediated by A Chimeric RNA.DNA Oligonucleotide", Proc Natl Acad Sci USA, 93, 2071-2076, (1996).
Cole-Strauss A. et al., "Correction of the Mutation Responsible For Sickle Cell Anemia by An RNA-DNA Oligonucleotide", Science, 273, 1386-1389, (1996).
Kren B. T. et al., "Targeted Nucleotide Exchange in the Alkaline Phosphatase Gene of HuH-7 cells Mediated By A Chimeric RNA/DNA Oligonucleotide", Hepatology, 27, 1462-1468, (1997).
Kmiec, E. B. et al., "The REC2 Gene Encodes the Homologous Pairing Protein of Ustilago Maydis", Mol Cell Biol, 14, 7163-7172, (1994).
Kmiec, E. B., "Genetic Manipulation in mammalian Cells Using an RNA/DNA Chimeric Oligonucleotide", Advanced Drug Delivery Reviews, 17, 333-340, (1995).
Roberts R. W. et al., "Stability and Properties of Double and Triple Helices: Dramatic Effects of RNA and DNA Backbone Composition", Science, 258, 1463-1466, (1992).
Cole-Strauss Allyson D.
Kmiec Eric B.
Elliott George C.
Hansburg Daniel
Thomas Jefferson University
Wang Andrew
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