INT283201

Context Info Confidence 0.00 First Reported 2009 Last Reported 2009 Negated 0 Speculated 0 Reported most in Body Documents 1 Total Number 1 Disease Relevance 0.56 Pain Relevance 0

endosome (Gnas) GTPase activity (Gnas) cytoplasm (Gnas) signal transducer activity (Gnas) cytosol (Gnas) signal transduction (Gnas)

Anatomy Link Frequency immune system 1 Gm4658 (Mus musculus) Gnas (Mus musculus)

Pain Link Frequency Relevance Heat beta blocker 1 5.00 agonist 1 5.00 fibrosis 1 5.00 depression 1 5.00 Inflammation 1 5.00 Pain 1 5.00

Disease Link Frequency Relevance Heat Pressure Volume 2 Under Development 1 97.84 Anaphylaxis 1 97.58 Muscular Dystrophy 43 90.16 Duchenne Muscular Dystrophy 4 16.04 Respiratory Failure 2 5.00 Depression 2 5.00 Stress 2 5.00 Fibrosis 1 5.00 Toxicity 1 5.00 Coronary Heart Disease 1 5.00

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Key:

Protein

Mutation

Event

Anatomy

Negation

Speculation

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Phosphorodiamidate morpholino oligomers (PMOs; figure 1) are non-toxic, and in the mdx mouse model of DMD they were the most effective oligomer chemistry for inducing exon skipping and restoring long-lasting (weeks) dystrophin expression after intravenous or intramuscular injection.21–24 PMOs, unlike other antisense oligonucleotides, are uncharged, not metabolised, and in preclinical or clinical studies were not associated with activation of the immune system, anaphylaxis, hypotension, or anti-arrhythmias.25 On the basis of these data, we have studied the safety and biochemical efficacy of AVI-4658, a PMO designed to target exon 51 that is delivered by intramuscular injection.

AVI-4658 Regulation (target) of exon in immune system associated with pressure volume 2 under development, anaphylaxis and muscular dystrophy

1) Confidence 0.00 Published 2009 Journal Lancet Neurol Section Body Doc Link PMC2755039 Disease Relevance 0.56 Pain Relevance 0

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