Improved pharmaceutical compositions for gene therapy

Details Improved pharmaceutical compositions for gene therapy Improved pharmaceutical compositions for gene therapy

C - Chemistry, Metallurgy

12

N

C12N 15/86 (2006.01) A61K 31/70 (2006.01) A61K 48/00 (2006.01) C07K 14/025 (2006.01) C07K 14/47 (2006.01) C07K 19/00 (2006.01)

Patent

CA 2224146

The invention is based on the discovery of a synthetic virus like particle containing a plurality of peptides capable of condensing nucleic acid and condensed nucleic acid. The plurality of peptides has a low polydispersion index and each peptide of said plurailty is a heteropeptide. The nucleic acid may encode sequences of therapeutic benefit. The synthetic virus like particle is self-assembling and may be designed so as to be capable of targeting a particular cell or tissue type and delivering nucleic acid to be incorporated into the chromosomal or extrachromosomal sequences of the target cells or tissues.

Cette invention est fondée sur la découverte d'une particule de synthèse du type virus contenant une pluralité de peptides capables de condenser de l'acide nucléique et de l'acide nucléique condensé. La pluralité de peptides présente un faible indice de polydispersion et chaque peptide est un hétéropeptide. L'acide nucléique peut coder des séquences possédant un intérêt thérapeutique. La particule de synthèse du type virus s'assemble elle-mème et peut être conçue de manière à pouvoir cibler une cellule ou un type de tissu particulier et apporter l'acide nucléique devant être incorporé dans les séquences chromosomiques ou extrachromosomiques des cellules ou des tissus ciblés.

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