Editing CCR5: a novel approach to HIV gene therapy

Advances in Experimental Medicine and Biology
Tatjana I CornuToni Cathomen

Abstract

Acquired immunodeficiency syndrome (AIDS) is a life-threatening disorder caused by infection of individuals with the human immunodeficiency virus (HIV). Entry of HIV-1 into target cells depends on the presence of two surface proteins on the cell membrane: CD4, which serves as the main receptor, and either CCR5 or CXCR4 as a co-receptor. A limited number of people harbor a genomic 32-bp deletion in the CCR5 gene (CCR5∆32), leading to expression of a truncated gene product that provides resistance to HIV-1 infection in individuals homozygous for this mutation. Moreover, allogeneic hematopoietic stem cell (HSC) transplantation with CCR5∆32 donor cells seems to confer HIV-1 resistance to the recipient as well. However, since Δ32 donors are scarce and allogeneic HSC transplantation is not exempt from risks, the development of gene editing tools to knockout CCR5 in the genome of autologous cells is highly warranted. Targeted gene editing can be accomplished with designer nucleases, which essentially are engineered restriction enzymes that can be designed to cleave DNA at specific sites. During repair of these breaks, the cellular repair pathway often introduces small mutations at the break site, which makes it possible to disrupt the...Continue Reading

Citations

May 14, 2016·Human Genetics·Ronald BenjaminMartin R Schiller
Dec 16, 2017·Cancer Metastasis Reviews·Xiuhui ChenAnil K Sood
Mar 6, 2016·Current Opinion in HIV and AIDS·Lachlan R GrayMelissa J Churchill
Jul 16, 2020·Cells·Antonio Carusillo, Claudio Mussolino
Dec 23, 2020·Human Gene Therapy·Tatjana I CornuToni Cathomen
Oct 30, 2020·Scientific Reports·Jenna Kropp SchmidtIgor I Slukvin

❮ Previous
Next ❯

Related Concepts

Related Feeds

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Blood And Marrow Transplantation

The use of hematopoietic stem cell transplantation or blood and marrow transplantation (bmt) is on the increase worldwide. BMT is used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells. Here is the latest research on bone and marrow transplantation.

Related Papers

Current Opinion in HIV and AIDS
Paula M Cannon, Carl H June
The New England Journal of Medicine
Lambros KordelasEssen HIV AlloSCT Group
Stem Cells and Development
Gabriel GonzalezRichard R Behringer
© 2022 Meta ULC. All rights reserved