Antisense oligonucleotides which combat aberrant splicing and me

Details Antisense oligonucleotides which combat aberrant splicing and me Antisense oligonucleotides which combat aberrant splicing and me

1995-01-26

1997-09-09

Rories, Charles C. P.

Chemistry: molecular biology and microbiology

Animal cell, per se ; composition thereof; process of...

Method of regulating cell metabolism or physiology

435 701, 435 703, 435 704, 536 231, 536 241, 536 243, 536 2431, 536 2433, 536 245, 514 44, C12N 506, C12N 516, C07H 2100, A61K 3170

Patent

active

056655932

ABSTRACT:
A method of combatting aberrant splicing in a pre-mRNA molecule containing a mutation is disclosed. When present in the pre-mRNA, the mutation causes the pre-mRNA to splice incorrectly and produce an aberrant mRNA or mRNA fragment different from the mRNA ordinarily encoded by the pre-mRNA. The method comprises hybridizing an antisense oligonucleotide to the pre-mRNA molecule to create a duplex molecule under conditions which permit splicing. The antisense oligonucleotide is one which does not activate RNase H, and is selected to block a member of the aberrant set of splice elements created by the mutation so that the native intron is removed by splicing and the first mRNA molecule encoding a native protein is produced. Oligonucleotides useful for carrying out the method are also disclosed.

REFERENCES:
patent: 5023243 (1991-06-01), Tullis
patent: 5149797 (1992-09-01), Pederson et al.
patent: 5220007 (1993-06-01), Pederson et al.
M. Kulka et al, PNAS, vol. 86, (issued Sep. 1989) pp. 6868-6872.
E. Rapaport et al., PNAS, vol. 89 (Sep. 1992) pp. 8577-8580.
T. Gura, Science, vol. 270 (Oct. 27, 1995) pp 575-577.
E. Uhlmann et al., Chem. Rev., vol. 90(4) (Jun. 1990) pp. 543-584.
J. Milligan et al., vol. 36 (14) (Jul. 9, 1993) pp. 1923-1937, J. Med. Chem.
C. Stein et al., vol. 261 (Aug 20, 1993) Science, pp. 1004-1012
B. Tseng et al., Cancer Gene Therapy, vol. 1 (1) (Mar., 1994) pp. 65-71.
R. Kole et al., Advance Drug Delivery Reviews 6, 271-286 (1991). Pre-mRNA splicing as a target for antisense obligonucleotides.
S.H. Munroe; Antisense RNA inhibits splicing of pre-mRNA in vitro; The Embo Journal 7, pp. 2523-2532 (1988).
P.J. Furdon and R. Kole; Inhibition of in Vitro Pre-mRNA Splicing by Antisense Deoxyoligonucleotide Analogues, Journal of Cellular Biochemistry Abstracts 18th Annual Meetings, (Mar. 27-Apr. 7, 1989).
Y. Zhuang and A.M. Weiner; A compensatory base change in human U2 snRNA can suppress a branch site mutation, Genes & Development 3, pp. 1545-1552 (1989).
V. Volloch et al; Inhibition of PRE-mRNA Splicing by Antisense RNA in Vitro: Effect of RNA Containing Sequences Complementary to Exons, Biochemical and Biophysical Research Communications 179, pp. 1593-1599 (1991).
C.F. Bennett et al; Cationic Lipids Enhance Cellular Uptake and Activity of Phosphorothioate Antisense Oligonucleotides Molecular Pharmacology 41, pp. 1023-1033 (1992).
Z. Dominski and R. Kole; Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides Proc. Natl. Acad. Sci. 90 pp. 8673-8677 (1993).
Kole et al. Advanced Drug Delivery Reviews 6: 271-286, 1991.
Dominski et al. Proc. Natl. Acad. Sci. 90: 8673-8677, 1993.
Ryder et al. Nuc. Acids Res. 18(24): 7373-7379, 1990.
Furdon et al. Nuc. Acids Res. 17(22): 9192-9204, 1989.
Munroe, S. EMBO Journal 7(8): 2523-2532, 1988.
Ruskin et al. Cell 43: 131-142, 1985.

Affiliated with

Also associated with

Rating

Antisense oligonucleotides which combat aberrant splicing and me does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Antisense oligonucleotides which combat aberrant splicing and me, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Antisense oligonucleotides which combat aberrant splicing and me will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-69222