Scientists are harnessing molecular-level “scissors” to cut DNA, enabling the removal or inactivation of undesirable segments.
The ultimate goal is to completely eradicate the virus from the body, although extensive research is required to ensure safety and efficacy.
While current HIV medications can halt the virus, they are unable to eliminate it entirely.
A team from the University of Amsterdam, presenting an abstract of their preliminary findings at a medical conference this week, emphasises that their work is currently only a “proof of concept” and is unlikely to result in an HIV cure in the near future.
Dr. James Dixon, an associate professor specialising in stem-cell and gene-therapy technologies at the University of Nottingham, concurs, stating that further scrutiny of the full findings is necessary.
He emphasises that extensive research is required to demonstrate that the results observed in cell assays can translate into effective therapy for the entire body.
Moreover,Dixon stresses that significant development is still required before this approach could have a meaningful impact on individuals living with HIV.
Other researchers also exploring the use of CRISPR to combat HIV such as Excision BioTherapeutics, reported that three HIV-positive volunteers experienced no serious side effects after 48 weeks of treatment.
However, Dr. Jonathan Stoye, a virus expert at the Francis Crick Institute in London, cautioned that eliminating HIV from all potential reservoirs in the body remains “extremely challenging.”
“Off-target effects of the treatment, with possible long-term side effects, remain a concern,” he stated.
“It therefore seems likely that many years will elapse before any such CRISPR- based therapy becomes routine – even assuming that it can be shown to be effective.”
HIV infects and targets immune system cells, utilising their internal machinery to replicate itself.
Despite effective treatment, some cells enter a resting, or latent, phase, retaining HIV DNA even when not actively generating new virus particles.
The majority of individuals with HIV require lifelong antiretroviral therapy, as discontinuation of these medications can lead to reactivation of dormant virus and resumption of complications.
A small number of cases have shown apparent “cures” following aggressive cancer therapy, which eradicated some infected cells. However, this approach would never be advocated solely for HIV treatment.
Breakthrough Technology – What is CRISPR Gene Editing
CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing tool that has garnered immense attention in the scientific community.
CRISPR-Cas9 allows scientists to precisely edit DNA sequences within living cells. The process relies on a protein called Cas9, which acts like molecular scissors, and a guide RNA that directs Cas9 to the specific target site in the genome.
Once at the target site, Cas9 cuts the DNA, enabling researchers to either insert, delete, or modify specific genetic information.
CRISPR technology holds promise for treating genetic disorders by correcting faulty genes. As well as the breakthrough application for HIV treatment, It could also potentially help cure diseases like cystic fibrosis, sickle cell anaemia, and muscular dystrophy.
CRISPR can also be applied in the agricultural industry. From enhancing crop resilience, improve yields, to making plants more resistant to pests and diseases.
The announcement from the team of scientist at Amsterdam University is yet another reminder of the ongoing efforts to use CRISPR to study gene function, model diseases, and develop new therapies.
In 2020, biochemists Jennifer Doudna and Emmanuelle Charpentier were awarded the Nobel Prize in Chemistry for their groundbreaking work on CRISPR-Cas9.
Ethical Dilemmas
While CRISPR offers immense potential, it also raises critical ethical questions. As CRISPR can edit embryos, and potentially allow parents to select specific traits for their children, the concept of “designer babies” has raised concerns amongst conservatives about playing God and exacerbating social inequalities.
CRISPR technology may also inadvertently modify unintended genes, leading to unforeseen consequences.
Some studies suggest that CRISPR could activate cancer-causing genes or disrupt tumour-suppressing genes as unexpected changes in chromosomes could occur during editing.
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