Gene therapy approaches to supply apolipoprotein a-i...

C - Chemistry – Metallurgy – 12 – N

Patent

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C12N 15/12 (2006.01) A61K 48/00 (2006.01) A61P 3/00 (2006.01) A61P 9/00 (2006.01) C07K 14/775 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2304814

The invention relates to genetic approaches to supply nucleotide sequences encoding modified forms of the native forms of apolipoprotein A-I (ApoA-I): mature ApoA-I, preproApoA-I and proApoA-I; including native ApoA-I modified to contain ApoA-I agonists, peptides which mimic the activity of ApoA-I; ApoA-I superagonists, peptides which exceed the activity of native ApoA-I; and modified native ApoA-I having one or more amphipathic helices replaced by the nucleotide sequences of one or more ApoA-I agonists; for the treatment of disorders associated with dyslipoproteinemia, including cardiovascular disease, atherosclerosis, restenosis, hyperlipidemia, and other disorders such as septic shock.

L'invention concerne des méthodes d'approche génétique servant à produire des séquences de nucléotides codant des formes modifiées des formes natives d'apolipoprotéine A-I (ApoA-I): ApoA-I mature, preproApoA-I et proApoA-I; y compris ApoA-I native modifiée de manière à contenir des agonistes de ApoA-I, des peptides imitant l'activité de ApoA-I; des superagonistes de ApoA-I, des peptides dépassant l'activité de ApoA-I native; ApoA-I native modifiée possédant une ou plusieurs hélices amphipathiques remplacées par les séquences de nucléotides d'un ou de plusieurs agonistes de ApoA-I; afin de traiter des troubles associés à la dyslipoprotéinémie, y compris des maladies cardio-vasculaires, l'athérosclérose, la resténose, l'hyperlipidémie et d'autres maladies, telles que le choc septique.

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