Gene therapy to the kidney using viral vectors.
Many pediatric diseases have reached a therapeutic plateau using currently available surgical and pharmacological approaches. Gene therapy has emerged as an exciting new technology to manipulate cells in the mammalian system, and in some cases, this method has achieved amazing therapeutic benefits. Compared to other organs, such as the brain, liver and lung, methods to genetically modify renal cells have received relatively little attention. The current review will discuss the challenges and important developments regarding gene therapy to the kidney, and relate the recent successes and failures to the future potential of gene therapy as a treatment modality in the context of pediatric disease.
Akbulut
Talha
T
Department of Physiology, Medical College of Wisconsin, Milwaukee, WI.
Park
Frank
F
Transplantation. 2000 Apr 27;69(8):1690-4
10836382
Nephron. 2000 Aug;85(4):307-16
10940741
Annu Rev Med. 1999;50:531-45
10073292
J Virol. 1999 Nov;73(11):9314-24
10516040
Nat Genet. 2003 Jul;34(3):297-302
12778174
JAMA. 2005 Sep 21;294(11):1373-9
16174696
Proc Natl Acad Sci U S A. 1999 Nov 9;96(23):13324-9
10557319
Gene Ther. 2003 Aug;10(15):1234-40
12858188
Mol Ther. 2001 Mar;3(3):284-92
11273769
Blood. 2004 Nov 1;104(9):2714-21
15231566
Gene Ther. 2007 Nov;14(22):1605-9
17898796
Gene Ther. 2003 Apr;10(7):559-68
12646861
Nat Med. 2001 Jan;7(1):33-40
11135613
Hum Gene Ther. 2005 Jan;16(1):17-25
15703485
Proc Natl Acad Sci U S A. 1994 May 10;91(10):4407-11
8183921
Cancer Res. 2002 Aug 1;62(15):4273-81
12154029
Exp Nephrol. 1993 Jan-Feb;1(1):41-8
8081951
Urol Res. 1999 Apr;27(2):97-102
10424390
J Virol. 2005 Jun;79(12):7478-91
15919903
Blood. 2002 Aug 1;100(3):813-22
12130491
Biochem Biophys Res Commun. 1992 Jul 15;186(1):129-34
1321600
Mol Ther. 2000 Jan;1(1):31-8
10933909
Pediatr Clin North Am. 2006 Aug;53(4):621-38
16872996
Kidney Int. 1994 Apr;45(4):1220-5
8007594
Nephron Exp Nephrol. 2004;96(4):e119-26
15122061
Gene Ther. 2004 Oct;11 Suppl 1:S10-7
15454952
Science. 1999 Dec 17;286(5448):2244-5
10636774
Proc Natl Acad Sci U S A. 2002 Sep 3;99(18):11854-9
12192090
Am J Physiol Heart Circ Physiol. 2007 Jul;293(1):H881-94
17322424
Mol Genet Metab. 2003 Sep-Oct;80(1-2):148-58
14567964
Hum Gene Ther. 2001 Jan 20;12(2):141-8
11177551
J Am Soc Nephrol. 2000 Apr;11(4):747-752
10752534
Mol Ther. 2000 Oct;2(4):374-80
11020353
Gene Ther. 1996 Apr;3(4):298-304
8732161
Mol Ther. 2003 Aug;8(2):180-7
12907140
Mol Biotechnol. 2007 Jul;36(3):184-204
17873406
Kidney Int. 2006 Feb;69(4):655-62
16395271
Annu Rev Genomics Hum Genet. 2001;2:177-211
11701648
Mol Ther. 2003 Jul;8(1):90-8
12842432
Mol Ther. 2006 Jul;14(1):45-53
16713360
EMBO J. 1997 May 1;16(9):2294-306
9171344
BJU Int. 2008 Feb;101(3):376-81
18184329
Kidney Int. 2002 Jan;61(1 Suppl):S16-23
11841607
Gene Ther. 1995 Mar;2(2):107-15
7719927
Hum Gene Ther. 2002 Feb 10;13(3):407-14
11860707
Science. 1996 Apr 12;272(5259):263-7
8602510
Pediatr Res. 2004 Jul;56(1):150-9
15128921
Transplantation. 1996 Mar 15;61(5):812-7
8607188
J Virol. 1998 Jan;72(1):415-9
9420240
Hum Gene Ther. 1997 Jul 1;8(10):1243-51
9215741
J Pharmacol Exp Ther. 2000 May;293(2):494-500
10773020
Pediatrics. 2000 Dec;106(6):1494-7
11099612
Transplantation. 1998 Jan 27;65(2):155-60
9458007
Nephrol Dial Transplant. 2001 Mar;16(3):608-14
11239040
Mol Cancer Ther. 2007 Oct;6(10):2728-36
17938266
Blood. 2006 Oct 15;108(8):2554-61
16788098
Cell. 2008 Feb 8;132(3):397-409
18267072
Kidney Int. 2000 May;57(5):1973-80
10792616
Exp Nephrol. 1997 Mar-Apr;5(2):132-6
9108995
Mol Ther. 2004 Jun;9(6):902-13
15194057
EMBO J. 1993 May;12(5):2099-108
8491198
Blood. 2000 Aug 1;96(3):1173-6
10910939
Mol Cell Biol. 1990 Aug;10(8):4239-42
2370865
J Clin Invest. 1993 Dec;92(6):2597-601
8254017
Hum Gene Ther. 2005 Jan;16(1):1-16
15703484
Blood. 2004 May 15;103(10):3700-9
14701690
N Engl J Med. 2003 Jan 16;348(3):255-6
12529469
Mol Ther. 2007 Sep;15(9):1647-54
17551506
J Am Soc Nephrol. 2003 Apr;14(4):947-58
12660329
Curr Gene Ther. 2007 Aug;7(4):261-71
17969559
Curr Gene Ther. 2005 Jun;5(3):285-97
15975006
Am J Physiol Renal Physiol. 2000 Jun;278(6):F949-53
10836982
Gene Ther. 1996 Jan;3(1):21-7
8929908
J Am Soc Nephrol. 1999 Sep;10(9):1908-15
10477142
Kidney Int. 2002 Jan;61(1 Suppl):S32-6
11841609
Gene Ther. 1999 Sep;6(9):1520-35
10490761
Clin J Am Soc Nephrol. 2008 Mar;3 Suppl 2:S68-75
18309005
Nephrol Dial Transplant. 2005 Jul;20(7):1385-91
15871986
Urology. 2003 Sep;62(3):559-65
12946777
Exp Nephrol. 1993 Jan-Feb;1(1):49-54
8081952
Curr Gene Ther. 2002 Sep;2(3):273-93
12189716
Nat Biotechnol. 1997 Sep;15(9):866-70
9306401
Science. 2003 Oct 17;302(5644):415-9
14564000
Am J Transplant. 2005 Jun;5(6):1194-203
15888023
Hum Gene Ther. 2000 Mar 1;11(4):621-7
10724040
Nat Biotechnol. 2002 Oct;20(10):999-1005
12244327
Cancer Gene Ther. 2001 Jul;8(7):529-36
11498775
Hum Gene Ther. 2001 Jul 20;12(11):1417-28
11485633
J Am Soc Nephrol. 2003 Mar;14(3):745-54
12595512
Science. 1995 Oct 20;270(5235):475-80
7570001
J Virol. 2005 Jan;79(1):214-24
15596817
Clin Exp Nephrol. 2006 Dec;10(4):229-35
17186326
Nat Biotechnol. 2002 Jan;20(1):53-7
11753362
Hum Gene Ther. 2004 Nov;15(11):1054-64
15610606