Creating genetic resistance to HIV

被引：34

作者：

Burnett, John C. ^{[1
]}

Zaia, John A. ^{[1
]}

Rossi, John J. ^{[1
]}

机构：

[1] City Hope Natl Med Ctr, Beckman Res Inst, Duarte, CA USA

来源：

CURRENT OPINION IN IMMUNOLOGY | 2012年 / 24卷 / 05期

关键词：

IMMUNODEFICIENCY-VIRUS TYPE-1; ZINC-FINGER NUCLEASES; MODIFIED T-CELLS; LENTIVIRAL VECTOR; CURRENT PROGRESS; CLINICAL-TRIAL; IN-VIVO; THERAPY; RIBOZYME; LYMPHOCYTES;

D O I：

10.1016/j.coi.2012.08.013

中图分类号：

R392 [医学免疫学]; Q939.91 [免疫学];

学科分类号：

100102 ;

摘要：

HIV/AIDS remains a chronic and incurable disease, in spite of the notable successes of combination antiretroviral therapy. Gene therapy offers the prospect of creating genetic resistance to HIV that supplants the need for antiviral drugs. In sight of this goal, a variety of anti-HIV genes have reached clinical testing, including gene-editing enzymes, protein-based inhibitors, and RNA-based therapeutics. Combinations of therapeutic genes against viral and host targets are designed to improve the overall antiviral potency and reduce the likelihood of viral resistance. In cell-based therapies, therapeutic genes are expressed in gene modified T lymphocytes or in hematopoietic stem cells that generate an HIV-resistant immune system. Such strategies must promote the selective proliferation of the transplanted cells and the prolonged expression of therapeutic genes. This review focuses on the current advances and limitations in genetic therapies against HIV, including the status of several recent and ongoing clinical studies.

引用

页码：625 / 632

页数：8

共 68 条

[41] HIV Restriction Factors and Mechanisms of Evasion [J].

Malim, Michael H. ;

Bieniasz, Paul D. .

COLD SPRING HARBOR PERSPECTIVES IN MEDICINE, 2012, 2 (05)

[42] Excision of HIV-1 Proviral DNA by Recombinant Cell Permeable Tre-Recombinase [J].

Mariyanna, Lakshmikanth ;

Priyadarshini, Poornima ;

Hofmann-Sieber, Helga ;

Krepstakies, Marcel ;

Walz, Nicole ;

Grundhoff, Adam ;

Buchholz, Frank ;

Hildt, Eberhard ;

Hauber, Joachim .

PLOS ONE, 2012, 7 (02)

[43] Autonomous Targeting of Infectious Superspreaders Using Engineered Transmissible Therapies [J].

Metzger, Vincent T. ;

Lloyd-Smith, James O. ;

Weinberger, Leor S. .

PLOS COMPUTATIONAL BIOLOGY, 2011, 7 (03)

[44] Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells [J].

Mitsuyasu, Ronald T. ;

Merigan, Thomas C. ;

Carr, Andrew ;

Zack, Jerome A. ;

Winters, Mark A. ;

Workman, Cassy ;

Bloch, Mark ;

Lalezari, Jacob ;

Becker, Stephen ;

Thornton, Lorna ;

Akil, Bisher ;

Khanlou, Homayoon ;

Finlayson, Robert ;

McFarlane, Robert ;

Smith, Don E. ;

Garsia, Roger ;

Ma, David ;

Law, Matthew ;

Murray, John M. ;

von Kalle, Christof ;

Ely, Julie A. ;

Patino, Sharon M. ;

Knop, Alison E. ;

Wong, Philip ;

Todd, Alison V. ;

Haughton, Margaret ;

Fuery, Caroline ;

Macpherson, Janet L. ;

Symonds, Geoff P. ;

Evans, Louise A. ;

Pond, Susan M. ;

Cooper, David A. .

NATURE MEDICINE, 2009, 15 (03) :285-292

[45] Potent inhibition of HIV-1 by TRIM5-cyclophilin fusion proteins engineered from human components [J].

Neagu, Martha R. ;

Ziegler, Patrick ;

Pertel, Thomas ;

Strambio-De-Castillia, Caterina ;

Gruetter, Christian ;

Martinetti, Gladys ;

Mazzucchelli, Luca ;

Gruetter, Markus ;

Manz, Markus G. ;

Luban, Jeremy .

JOURNAL OF CLINICAL INVESTIGATION, 2009, 119 (10) :3035-3047

[46] Long-term Regulation of Genetically Modified Primary Hematopoietic Cells in Dogs [J].

Okazuka, Kiyoshi ;

Beard, Brian C. ;

Emery, David W. ;

Schwarzwaelder, Kerstin ;

Spector, Michele R. ;

Sale, George E. ;

von Kalle, Christof ;

Kiem, Hans-Peter ;

Blau, C. Anthony .

MOLECULAR THERAPY, 2011, 19 (07) :1287-1294

[47] Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases [J].

Perez, Elena E. ;

Wang, Jianbin ;

Miller, Jeffrey C. ;

Jouvenot, Yann ;

Kim, Kenneth A. ;

Liu, Olga ;

Wang, Nathaniel ;

Lee, Gary ;

Bartsevich, Victor V. ;

Lee, Ya-Li ;

Guschin, Dmitry Y. ;

Rupniewski, Igor ;

Waite, Adam J. ;

Carpenito, Carmine ;

Carroll, Richard G. ;

Orange, Jordan S. ;

Urnov, Fyodor D. ;

Rebar, Edward J. ;

Ando, Dale ;

Gregory, Philip D. ;

Riley, James L. ;

Holmes, Michael C. ;

June, Carl H. .

NATURE BIOTECHNOLOGY, 2008, 26 (07) :808-816

[48] Ribozyme therapy for HIV infection [J].

Rossi, JJ .

ADVANCED DRUG DELIVERY REVIEWS, 2000, 44 (01) :71-78

[49] Deriving four functional anti-HIV siRNAs from a single Pol III-generated transcript comprising two adjacent long hairpin RNA precursors [J].

Saayman, Sheena ;

Arbuthnot, Patrick ;

Weinberg, Marc S. .

NUCLEIC ACIDS RESEARCH, 2010, 38 (19) :6652-6663

[50] Sequence homology required by human immunodeficiency virus type 1 to escape from short interfering RNAs [J].

Sabariegos, R ;

Giménez-Barcons, M ;

Tàpia, N ;

Clotet, B ;

Martínez, MA .

JOURNAL OF VIROLOGY, 2006, 80 (02) :571-577

← 1 2 3 4 5 6 7 →