Zbigniew T Dominski

US Patent:

56272740, May 6, 1997

Filed:

May 30, 1995

Appl. No.:

8/453224

Inventors:

Ryszard Kole - Chapel Hill NC
Zbigniew Dominski - Chapel Hill NC

Assignee:

The University of North Carolina at Chapel Hill - Chapel Hill NC

International Classification:

C07H 2100
A61K 4800
C12Q 168

US Classification:

536 231

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.

US Patent:

59768797, Nov 2, 1999

Filed:

Apr 30, 1999

Appl. No.:

9/302390

Inventors:

Ryszard Kole - Chapel Hill NC
Zbigniew Dominski - Chapel Hill NC

Assignee:

The University of North Carolina at Chapel Hill - Chapel Hill NC

International Classification:

C12Q 168
C12N 1585
C02H 2104

US Classification:

435375

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.

US Patent:

59168080, Jun 29, 1999

Filed:

Feb 19, 1997

Appl. No.:

8/802384

Inventors:

Ryszard Kole - Chapel Hill NC
Zbigniew Dominski - Chapel Hill NC

Assignee:

The University of North Carolina at Chapel Hill - Chapel Hill NC

International Classification:

C12Q 168
C07H 2100

US Classification:

435375

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.