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Home Gene Therapy Antisense-inhibition-of-PEPCK-mitochondrial-expression

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 Antisense inhibition of PEPCK-mitochondrial expression

Details
Inventors: McKay, Robert; Butler, Madeline M.; Cowsert, Lex M.;
Assignee: Isis Pharmaceuticals Inc. (Carlsbad, CA)
Primary Examiner: LeGuyader; John L.
Assistant Examiner: Hickey; Karen A.
Attorney, Agent or Firm: Law Offices of Jane Massey Licata

Antisense compounds, compositions and methods are provided for modulating the expression of PEPCK-mitochondrial. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding PEPCK-mitochondrial. Methods of using these compounds for modulation of PEPCK-mitochondrial expression and for treatment of diseases associated with expression of PEPCK-mitochondrial are provided.

DETAILED DESCRIPTION OF THE INVENTION The present invention employs oligomeric antisense compounds, particularly oligonucleotides, for use in modulating the function of nucleic acid molecules encoding PEPCK-mitochondrial, ultimately modulating the amount of PEPCK-mitochondrial produced.
This is accomplished by providing antisense compounds which specifically hybridize with one or more nucleic acids encoding PEPCK-mitochondrial.
As used herein, the terms "target nucleic acid" and "nucleic acid encoding PEPCK-mitochondrial" encompass DNA encoding PEPCK-mitochondrial, RNA (including pre-mRNA and mRNA) transcribed from such DNA, and also cDNA derived from such RNA.
The specific hybridization of an oligomeric compound with its target nucleic acid interferes with the normal function of the nucleic acid.
This modulation of function of a target nucleic acid by compounds which specifically hybridize to it is generally referred to as "antisense".
The functions of DNA to be interfered with include replication and transcription.
The functions of RNA to be interfered with include all vital functions such as, for example, translocation of the RNA to the site of protein translation, translation of protein from the RNA, splicing of the RNA to yield one or more MRNA species, and catalytic activity which may be engaged in or facilitated by the RNA.
The overall effect of such interference with target nucleic acid function is modulation of the expression of PEPCK-mitochondrial.
In the context of the present invention, "modulation" means either an increase (stimulation) or a decrease (inhibition) in the expression of a gene.
In the context of the present invention, inhibition is the preferred form of modulation of gene expression and mRNA is a preferred target.
It is preferred to target specific nucleic acids for antisense.
"Targeting" an antisense compound to a particular nucleic acid, in the context of this invention, is a multistep process.
The process usually begins with the identification of a nucleic acid sequence whose function is to be modulated



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