Patent number 8557875 is assigned to
The following quote was obtained by the news editors from the background information supplied by the inventors: "Viral vectors, known as gene transfer agents with high efficiency, are used for genetic therapies and the like. However, there are reports that viral vectors have severe side effects leading to death in clinical applications. Hence, a gene transfer agent with high safety has been demanded.
"Although non-viral vectors show higher safety than that of viral vectors, non-viral vectors have a drawback in that their gene transfer efficiency is lower.
"The inventor of the present invention has already developed, as an excellent gene transfer agent composition that can solve the above-mentioned problem, a gene transfer agent composition containing a polyamidoamine dendron (Patent Literature 1)."
In addition to the background information obtained for this patent, NewsRx journalists also obtained the inventor's summary information for this patent: "Technical Problem
"An object of the present invention is to provide a gene transfer agent, a gene transfer kit, and a gene transfer method excellent in safety and transfer efficiency. In particular, another object of the present invention is to provide an improved gene transfer agent composition containing polyamidoamine dendron.
"Solution to Problem
"The present invention provides the following gene transfer agent, gene transfer kit, and gene transfer method.
"Item 1. A gene transfer agent composition comprising a compound represented by any one of the following formulae DL-G1 to DL-G4: R.sup.1R.sup.2NX(XH.sub.2).sub.2; DL-G1: R.sup.1R.sup.2NX(X(XH.sub.2).sub.2).sub.2; DL-G2: R.sup.1R.sup.2NX(X(X(XH.sub.2).sub.2).sub.2).sub.2; DL-G3: and R.sup.1R.sup.2NX(X(X(X(XH.sub.2).sub.2).sub.2).sub.2).sub.2, DL-G4:
"(X represents --CH.sub.2CH.sub.2CONHCH.sub.2CH.sub.2N--)
"R.sup.1 represents an unsaturated long-chain aliphatic group; and
"R.sup.2 represents an unsaturated long-chain aliphatic group or a saturated long-chain aliphatic group in the above-mentioned formulae.
"Item 2. The gene transfer agent composition according to Item 1, in which the long-chain aliphatic group comprises an aliphatic group having 10 to 22 carbon atoms.
"Item 3. The gene transfer agent composition according to Item 1, in which the unsaturated long-chain aliphatic group is a hexadecenyl group, an octadecenyl group, an octadecadienyl group, an octadecatrienyl group, an icosatrienyl group, an icosatetraenyl group, an octadecatrienyl group, an icosapentaenyl group, or a docosahexaenyl group. Item 4. The gene transfer agent composition according to any one of Items 1 to 3, further comprising a phospholipid. Item 5. The gene transfer agent composition according to Item 4, in which the phospholipid comprises DOPE. Item 6. A method of transferring a gene into a cell, comprising applying the gene transfer agent composition according to any one of Items 1 to 5 and a gene to a cell in vitro or in vivo (provided that a human is excluded). Item 7. A gene transfer kit, including the gene transfer agent composition according to any one of Items 1 to 5.
"Advantageous Effects of Invention
"According to the present invention, it is possible to provide a gene transfer agent with significantly improved gene transfer efficiency and significantly reduced cytotoxicity as compared to a conventional gene transfer agent. Therefore, it is possible to provide a gene transfer technology that is excellent in both transfer efficiency and safety, and that can find a wide range of practical uses."
URL and more information on this patent, see:
Keywords for this news article include: Biotechnology, Viral, Virus, Gene Therapy, Bioengineering,
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