Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

In this small study, investigators genetically disrupted the HIV receptor CCR5 in CD4 T cells of aviremic persons who were infected with HIV. After reinfusion and during viremia, the modified cells declined at a significantly slower rate than did the unmodified cells. The ability to make site-specif...

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Bibliographic Details
Published in:	The New England journal of medicine Vol. 370; no. 10; pp. 901 - 910
Main Authors:	Tebas, Pablo, Stein, David, Tang, Winson W, Frank, Ian, Wang, Shelley Q, Lee, Gary, Spratt, S. Kaye, Surosky, Richard T, Giedlin, Martin A, Nichol, Geoff, Holmes, Michael C, Gregory, Philip D, Ando, Dale G, Kalos, Michael, Collman, Ronald G, Binder-Scholl, Gwendolyn, Plesa, Gabriela, Hwang, Wei-Ting, Levine, Bruce L, June, Carl H
Format:	Journal Article
Language:	English
Published:	Waltham, MA Massachusetts Medical Society 06-03-2014
Subjects:	Adult Allergies Antiretroviral Therapy, Highly Active Biological and medical sciences Blood Transfusion, Autologous CCR5 protein CD4 antigen CD4-Positive T-Lymphocytes - chemistry CD4-Positive T-Lymphocytes - transplantation Chronic infection Combined Modality Therapy Deoxyribonucleic acid DNA DNA repair DNA, Viral - blood Female General aspects Genetic Therapy - adverse effects Genetic Therapy - methods Genome editing Genomes Highly active antiretroviral therapy HIV HIV - genetics HIV - isolation & purification HIV Infections - drug therapy HIV Infections - immunology HIV Infections - therapy Human immunodeficiency virus Human viral diseases Humans Immune reconstitution Immunodeficiencies Immunodeficiencies. Immunoglobulinopathies Immunopathology Infectious diseases Leukocytes (mononuclear) Lymphocyte Count Lymphocyte Transfusion Lymphocytes Lymphocytes T Male Medical sciences Middle Aged Nuclease Patients Peripheral blood Receptors, CCR5 - genetics Rectum - immunology Ribonucleic acid RNA RNA, Viral - blood Viral diseases Viral diseases of the lymphoid tissue and the blood. Aids Viral Load Viremia Zinc finger proteins Immunopathology Retroviridae AIDS Immune deficiency Lentivirus Virus Infection Medicine CCR5 chemokine receptor Immunological investigation Viral disease T-Lymphocyte Human immunodeficiency virus
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Summary:	In this small study, investigators genetically disrupted the HIV receptor CCR5 in CD4 T cells of aviremic persons who were infected with HIV. After reinfusion and during viremia, the modified cells declined at a significantly slower rate than did the unmodified cells. The ability to make site-specific modifications to (or “edit”) the human genome has been an objective in medicine since the recognition of the gene as the basic unit of heredity. 1 , 2 The challenge of genome editing is the ability to generate a single double-strand break at a specific point in the DNA molecule. Numerous agents, including meganucleases, oligonucleotides that form DNA triplexes, and peptide nucleic acids, have been tested and shown to be limited by inefficiency. 3 – 5 Another class of reagents, the zinc-finger nucleases (ZFNs), have proved versatile for genome editing, and the use of ZFNs is now well established . . .
Bibliography:	ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-1 content type line 23
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa1300662