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Isis Pharmaceuticals, Inc. Message Board

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  • dinepat203 dinepat203 Dec 11, 2012 1:36 PM Flag

    Why pharma Giants wants ISIS? GSK,SANOFI-GENZYME,BIIB, ASTRAZENICA...wants newer antisense molecules..,

    Antisense therapy, a magic bullet for the treatment of various diseases: Present and future prospectAntisense drugs are a new generation of gene-silencing
    therapeutic agents with potential for targeted causal
    treatment of as yet incurable diseases. Antisense therapy
    is a form of treatment for genetic disorders or infections.
    When the genetic sequence of a particular gene is known
    to be causative of a particular disease, it is possible to
    synthesize a strand of nucleic acid (DNA, RNA or a
    chemical analogue) that will bind to the messenger RNA
    (mRNA) produced by that gene and inactivate it,
    effectively turning that gene "off". This is because mRNA
    has to be single stranded for it to be translated.
    Alternatively, the strand might be targeted to bind a
    splicing site on pre-mRNA and modify the exon content of
    an mRNA
    This synthesized nucleic acid is termed an "anti-sense"
    oligonucleotide because its base sequence is
    complementary to the gene's messenger RNA (mRNA),
    Abbreviations: ASO, Antisense oligonucleotides; CFTR, cystic
    fibrosis transmembrane conductance regulator; ODN, antisense
    oligodesoxynucleoties.
    which is called the "sense" sequence (so that a sense
    segment of mRNA " 5'-AAGGUC-3' " would be blocked by
    the anti-sense mRNA segment " 3'-UUCCAG-5' ").
    Antisense drugs are being researched to treat cancers
    including lung cancer, colorectal carcinoma, pancreatic
    carcinoma, malignant glioma and malignant melanoma,
    diabetes, Duchenne muscular dystrophy and diseases
    such as asthma and arthritis with an inflammatory
    component (Agrawal et al., 1995; 2000). Some of the
    examples of antisense drug like fomivirsen (marketed as
    Vitravene), has been approved by the US Food and Drug
    Administration (FDA) as a treatment for cytomegalovirus
    retinitis. Mipomersen (ISIS 301012) inhibits human
    apolipoprotein (apo) B-100 synthesis and lowers
    circulating apoB and low-density lipoprotein cholesterol
    levels.

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