Ribozyme-mediated gene replacement

Details Ribozyme-mediated gene replacement Ribozyme-mediated gene replacement

1997-05-14

2000-06-20

Brusca, John S.

Chemistry: molecular biology and microbiology

Vector, per se

435 6, 435 9331, 536 231, 536 241, 536 245, C07H 2104, C12N 1585, C12Q 168

Patent

active

060777052

ABSTRACT:
Methods of gene therapy, particular ribozyme-mediated gene replacement methods, are disclosed. Method of treating patients suffering from a disease associated with expression of an abnormal form of a gene, such as alpha-1 antitrypsin mutations, are disclosed. The methods comprise the steps of administering to such a patient a nucleic acid construct encoding a ribozyme and a nucleic acid construct comprising a ribozyme resistant gene encoding a wild type form of the gene product. Recombinant vectors and pharmaceutical compositions for practicing the methods are disclosed.

REFERENCES:
patent: 5399684 (1995-03-01), Davie et al.
Cai et al "Suppresion of Lung Cancer Cell Growth by Ribozym-Mediated Modification of p53 pre-mRNA" Cancer Gene Therapy vol. 2(3):199-205, Sep. 1995.
Bollen et al., 1983, DNA 2(4),255-64.
Chomczynski, P, et al., 1987, Anal. Biochem., 162:156-159.
Davis, E.S. et al., 1990, J. Biol. Chem., 265: 22153-22158.
Denman, R.B. 1993, Biotechniques, 15:1090-1094.
Frizell, E. et al., 1995, Hepatology, 21:847-854.
Lee, S.W. et al., 1994 J. Virol. 68:8254-8264.
Mol. Cell. Biol.,1986 6: 2895-2902.
Scharfmann, R. et al., 1991 Proc. Natl. Acad. Sci. USA 88:4624-4630.
Strayer, D.S. and J. Milano 1996 Gene Therapy 3:581-587.
Strayer, D.S. 1996 J. Biol. Chem. 27:24741-24746.
Yun-Tso, J. et al., 1985, Nucl. acid. Res., 13, 2485-2502.
Pierce et al "Construction of a Directed Hammerhead Ribozyme Library: Towards the Identification of Optimal Target Sites for Antisense-Mediated Gene Inhibition" NAR vol. 26(22):5093-5101, 1998.

Affiliated with

Also associated with

Rating

Ribozyme-mediated gene replacement does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Ribozyme-mediated gene replacement, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Ribozyme-mediated gene replacement will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-1851079