Cell therapy using CRISPR for HIV treatment: The first steps?

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In the last few years, CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, has emerged as a powerful technique for gene editing and regulation with significant implications for gene therapy.

Since a seminal paper Science paper in 2007 showed that gene modifications via CRISPR may impart virus resistance to bacteria, genome engineering efforts via CRISPR have fluorished.

CRISPR is, in simpler terms, a set of genetic sequences that were discovered in bacteria in the 1980s. CRISPR allows researchers to make precise changes to the genomes of both human and non-human cells, but unlike other genome editing approaches, CRISPR enables doing so in a less technically challenging way.

Recently, using genome editing for therapeutic applications has resulted in a influx of papers that have tackled significant medical problems: one that has garnered a lot of traction is using CRISPR as a new way to treat HIV. In a recent paper published in PNAS, Kamela Khalili and colleagues at NIAID showed that gene editing could be used to effectively erase HIV genes from infected cells and thereby prevent new infection.

This week, a paper by Chad Coward’s lab at Harvard extended the use of CRISPR by demonstrating a potentially new treatment for HIV for use in cell therapy.

The paper, titled “Efficient Ablation of Genes in Human Hematopoietic Stem and Effector Cells using CRISPR/Cas9” was published this month in CellStemCell.

The authors demonstrated that they could efficiently erase the CCR5 gene receptor – which acts as the “gatekeeper” for HIV to infect T cells – from hematopoietic stem and progenitor cells (HSPCs). By doing so, the team generated gene-edited stem cells that did not have the CCR5 gene, which could then be introduced into HIV patients via bone marrow transplantation and ultimately generate HIV-resistant immune systems.

While the work is potentially revolutionary and the approach efficient, the authors responsibly point out that the results are preliminary, and while clinical studies are being planned, mouse studies need to follow first before broader statements about this as a viable therapy can be made.

Nevertheless, the paper is an interesting and compelling read – find it here.

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