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Reagent for introduction of protein or gene

Foreign code F110003065
File No. 134-US
Posted date May 25, 2011
Country United States of America
Application number 31264807
Gazette No. 20100291672
Gazette No. 8338643
Date of filing Nov 26, 2007
Gazette Date Nov 18, 2010
Gazette Date Dec 25, 2012
International application number JP2007073266
International publication number WO2008062911
Date of international filing Nov 26, 2007
Date of international publication May 29, 2008
Priority data
  • P2006-317841 (Nov 24, 2006) JP
  • 2007WO-JP73266 (Nov 26, 2007) WO
Title Reagent for introduction of protein or gene
Abstract (US8338643)
The present invention provides a reagent for introducing a protein or gene into a cell.
The reagent of the present invention is, for example, a reagent for introducing a protein or gene into a cell, which comprises a composition comprising a cationic amino acid type lipid represented by the following formula (I)-1: (wherein in formula (I)-1: L is a single bond, —CONH—, or —S—S—; M1 is —(CH2)k— or —(CH2CH2O)k— (wherein k is an integer between 0 and 14); and m1 and m2 are each independently an integer between 11 and 21 (in this regard, when providing a reagent for introducing a gene into a cell, the case where both m1 and m2 are 15 is excluded)).
Scope of claims [claim1]
1. A reagent for introducing a protein into a cell, which comprises a composition comprising a cationic amino acid type lipid represented by the following formula (I)-1:

wherein: L is a single bond, -- CONH -- , or -- S -- S -- ; M1 is -- (CH2)k -- or -- (CH2CH2O)k -- ; k is an integer between 1 and 11; and m1 and m2 are each independently an integer between 11 and 21.
[claim2]
2. A reagent for introducing a gene into a cell, which comprises a composition comprising a cationic amino acid type lipid represented by the following formula (I)-1:

wherein: L is a single bond, -- CONH -- , or -- S -- S -- ; M1 is -- (CH2)k -- or -- (CH2CH2O)k -- ; k is an integer between 1 and 11; and m1 and m2 are each independently an integer between 11 and 21; with the proviso that both m1 and m2 are not an integer of 15.
[claim3]
3. The reagent according to claim 1 or 2, wherein the composition further comprises at least one substance selected from the group consisting of diacylphosphatidylcholine, cholesterol, and an amphipathic molecule to which a polyethylene glycol chain is bound.
[claim4]
4. The reagent according to claim 3, wherein one or two acyl chains of diacylphosphatidylcholine is an oleoyl group.
[claim5]
5. The reagent according to claim 1 or 2, wherein the composition is a molecular assembly dispersed in an aqueous medium.
[claim6]
6. The reagent according to claim 5, wherein the molecular assembly forms a bilayer membrane vesicle structure, and wherein an inner aqueous phase thereof includes a protein or gene.
[claim7]
7. The reagent according to claim 1 or 2, wherein the composition is a dry powder product.
[claim8]
8. The reagent according to claim 7, wherein the dry powder product can form a bilayer membrane vesicle structure including a protein or gene when dispersed in an aqueous medium.
[claim9]
9. The reagent according to claim 1 or 2, wherein the cell is: a cell constituting a part of body tissue; plasma, serum or blood; or a cell cultured in a medium containing a part of components thereof.
[claim10]
10. A kit for introducing a protein or gene into a cell, which comprises the reagent according to claim 1 or 2.
[claim11]
11. A cationic amino acid type lipid represented by the following formula (I)-3:

wherein: M2 is -- (CH2)k' -- or -- (CH2CH2O)k' -- ; k' is an integer between 1 and 14; and p1 and p2 are each independently an integer between 11 and 21.
[claim12]
12. A reagent for introducing a protein into a cell, which comprises a composition comprising a cationic amino acid type lipid represented by the following formula (I)-1:

wherein: L is -- CONH -- , or -- S -- S -- ; M1 is -- (CH2)k -- or -- (CH2CH2O)k -- ; k is an integer between 2 and 14; and m1 and m2 are each independently an integer between 11 and 21.
[claim13]
13. A reagent for introducing a gene into a cell, which comprises a composition comprising a cationic amino acid type lipid represented by the following formula (I)-1:

wherein: L is -- CONH -- , or -- S -- S -- ; M1 is -- (CH2)k -- or -- (CH2CH2O)k -- ; k is an integer between 2 and 14; and m1 and m2 are each independently an integer between 11 and 21; with the proviso that both m1 and m2 are not an integer of 15.
[claim14]
14. The reagent according to claim 12 or 13, wherein the composition further comprises at least one substance selected from the group consisting of diacylphosphatidylcholine, cholesterol, and an amphipathic molecule to which a polyethylene glycol chain is bound.
[claim15]
15. The reagent according to claim 14, wherein one or two acyl chains of diacylphosphatidylcholine is an oleoyl group.
[claim16]
16. The reagent according to claim 12 or 13, wherein the composition is a molecular assembly dispersed in an aqueous medium.
[claim17]
17. The reagent according to claim 16, wherein the molecular assembly forms a bilayer membrane vesicle structure, and wherein an inner aqueous phase thereof includes a protein or gene.
[claim18]
18. The reagent according to claim 12 or 13, wherein the composition is a dry powder product.
[claim19]
19. The reagent according to claim 18, wherein the dry powder product can form a bilayer membrane vesicle structure including a protein or gene when dispersed in an aqueous medium.
[claim20]
20. The reagent according to claim 12 or 13, wherein the cell is: a cell constituting a part of body tissue; plasma, serum or blood; or a cell cultured in a medium containing a part of components thereof.
[claim21]
21. A kit for introducing a protein or gene into a cell, which comprises the reagent according to claim 12 or 13.
[claim22]
22. The reagent according to claim 1, wherein k is an integer between 3 and 5.
[claim23]
23. The reagent according to claim 2, wherein k is an integer between 3 and 5.
  • Inventor, and Inventor/Applicant
  • TAKEOKA SHINJI
  • TAKEDA NAOYA
  • KURUMIZAKA HITOSHI
  • SAKANE ISAO
  • IKEGAYA NAMIKO
  • OBATA YOSUKE
  • SAITO SYUNSUKE
  • WASEDA UNIVERSITY
IPC(International Patent Classification)
U.S. Cl./(Sub)
  • C07C237/08
  • C07C237/12
  • C07C237/22
  • C07K005/02D
  • C07K005/06B
  • C07K005/08B
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