摘要:
Disclosed is expression of zeta negative and zeta positive nucleic acids or nucleic acid complexes using a dystrophin gene in a process for providing nucleic acid expression in a striated (skeletal or cardiac) muscle cell for the purpose of providing a change to the endogenous properties of the cell for cells affected by muscular dystrophy.
摘要:
Methods are described for modifying nucleic acids to facilitate delivery of the nucleic acids to cells. Compounds which interact with of modify nucleic acids are interacted with the nucleic acids within reverse micelles.
摘要:
A process and compound wherein nucleic acids can be modified with a host of molecules and maintain their ability to be expressed. A modifying chemical attachment of polyions to polynucleotides can be used to facilitate the change of tertiary structure of the nucleic acid and in some cases condensation of nucleic acids into smaller, charged particles useful in delivering the nucleic acid to a cell.
摘要:
Compounds and methods are provided for a single-pot covalent attachment of a label to nucleic acids comprising forming a covalently attachable labeling reagent for alkylating the molecule. Then, combining the covalently attachable labeling reagent with a mixture containing the molecule, under conditions wherein the labeling reagent has reactivity with the molecule thereby forming a covalent bond.
摘要:
A method of forming polymers in the presence of nucleic acid using template polymerization. These methods can be used for the delivery of nucleic acids, for condensing the nucleic acid, for forming nucleic acid binding polymers, for forming supramolecular complexes containing nucleic acid and polymer, and for forming an interpolyelectrolyte complex.
摘要:
Polyampholyte are able to condense nucleic acid to form small complexes which can be utilized in the delivery of nucleic acid to mammalian cells. The polyampholytes can be formed prior to interaction with nucleic acid or they can be formed in the presence of nucleic acid. Stabilized polycation/nucleic acid complexes can be modified to reduce the positive charge of the polycation and add targeting ligands without destabilizing the complex. The resultant particles retain their small size and are more effective in delivery of nucleic acid to cells in vivo.
摘要:
A method of forming polymers in the presence of nucleic acid using template polymerization. Also, a method of having the polymerization occur in heterophase systems. These methods can be used for the delivery of nucleic acids, for condensing the nucleic acid, for forming nucleic acid binding polymers, for forming supramolecular complexes containing nucleic acid and polymer, and for forming an interpolyelectrolyte complex.
摘要:
An polyampholyte is utilized in a condensed polynucleotide complex for purposes of nucleic acid delivery to a cell. The complex can be formed with an appropriate amount of positive and/or negative charge such that the resulting complex can be delivered to the extravascular space and may be further delivered to a cell.
摘要:
Described is a deliverable composition with low toxicity comprising an amphipathic compound, a polycation, and a siRNA. The composition may be used in the process of delivering a siRNA to an animal cell or more particularly, a mammal cell.
摘要:
A process is provided to deliver polynucleotide-based gene expression inhibitors to cells in a mammal in vivo for the purpose of inhibiting gene expression in the cells. Inhibition is sequence-specific and relies on sequence similaroty of the polynucleotide-based gene expression inhibitor and the target nucleic acid molecule. Delivery of the polynucleotide-based gene expression inhibitor can enhance the efficacy of co-delivered small molecule drugs.