If you have been following the world of genomic engineering, you may be familiar with the recent discoveries by UC Berkeley scientist Jennifer Doudna and Feng Zhang, faculty member at MIT and core member of the Broad Institute.
They have invented a series of gene splicing technology tools collectively known as CRISPR-Cas9.
This new approach to gene splicing is so revolutionary that there’s a major argument brewing between Berkeley and MIT over which of these two famed researchers can claim to have made the discovery first.
We’re going to skip past the argument over patent rights (which will determine who can collect millions of dollars in royalties from the discovery) and move on to the theory behind CRISPR-Cas9 — and the implications for ongoing HIV research and the potential for novel new HIV treatments.
This discovery started with researchers studying the patterns of DNA in bacteria; they were paying particular attention to the ways that bacteria use their DNA sequences to protect themselves from other viruses and phages. The researches noticed that were able to identify repeated DNA patterns.
As you might imagine, DNA often uses these type of repeating genetic patterns, which, for our simplified discussion, we will call words. Scientists then discovered that they can actually cut the bacteria’s genomic sequences at specific at these specific word locations. The result is the bacteria will try to repair itself. Sometimes this is successful, and sometimes it leads to an unpredictable genetic mutation.
Later scientists realized that they could employ a helper gene as a template to insert new genetic material where they made the cut at the word sequence. In layman’s terms what this means is that scientists now have the capacity to cut out genetic sequences, copy them and then paste them in new locations. In other words, CRISPR-Cas9 is comparable to a word processor for genomic sequences.
UC Berkeley Scientist Jennifer Doudna explains advances in Genomic Engineering using CRISPR-Cas9 technology.
Because this CRISPR-Cas9 enables inexpensive and easily accessible genomic sequences editing, you might have concerns about whether the widespread adoption of these techniques across laboratories across the world could create problems.
You are not alone. Bio-ethicists around the world of sound the alarm and there is widespread agreement that editing human genomes should be delayed indefinitely until the risks are understood and considered to outweigh the benefits.
There has not been universal agreement on this point. Genetic laboratory researchers at the Sun Yat-sen University in Guangzhou China attempted to modify human blood DNA in an attempt to cure a genetic blood disease (Beta thalassemia), a hereditary disease affecting hemoglobin. This month, 150 scientists, legal experts and entrepreneurs met at Harvard Medical School in secret to discuss how to manage human gene manipulation.
Implications of CRISPR-Cas9 for Future HIV Treatment
Recently, researchers at Temple University in Philadelphia have investigated ways to eliminate HIV-1 virus genomes from human T-lymphoid Cells (commonly known as T cells) using CRISPR-Cas9 genetic editing techniques.
The intent of the scientists is to pursue a cure strategy for HIV-1 infection that includes directly eliminating the viral genome in the majority of HIV-1 infected cells, including the CD4+ T cells. The advantage of this approach is it will protect cells from future infection with little or no harm to the host. The results of the experiments in the laboratory offer the promise of a potential therapeutic approach to eradicate HIV-1 virus infection from T reservoir cells of host patients once and for all — in order to prevent reemergence of AIDS in patients.
To confirm, these experiments were done in the laboratory on cells, taken outside the body; they were not performed on living patients.
However, the company organized with the exclusive rights to commercialize the technology, Excision Biotherapeutics, was quoted in a statement saying that it is a major step forward in eradicating HIV infection in humans using CRISPR-Cas9 genomic editing tools.
We will continue to provide you with updates as these stories develop.