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CANADA —Researchers from the University of Waterloo in Canada have unveiled a promising new strategy in combating HIV through gene therapy.
Published in the Journal of Controlled Release, their study outlines the development of an innovative RNA nanomedicine capable of significantly curtailing HIV replication within cells.
Despite the effectiveness of antiretroviral therapy (ART) in managing the disease, HIV remains incurable, and without intervention, it can progress to AIDS over time.
In response, the researchers devised a nanomedicine loaded with small interfering RNAs (siRNAs) designed to target specific genes crucial for HIV replication, resulting in a remarkable 73% reduction in viral activity within cells.
Moreover, the nanomedicine tackles an additional hurdle by addressing HIV’s ability to suppress autophagy, the body’s natural defense mechanism against viruses and bacteria.
By targeting a viral gene known as Nef, which inhibits autophagy, the nanomedicine enables infected cells to regain their ability to combat the virus.
The study introduces a ‘dual preventive strategy’. Firstly, by targeting a host gene called CCR5, which the virus exploits to enter cells, the nanomedicine aims to impede HIV from binding and infecting healthy cells. Secondly, it targets the Nef gene to restore autophagy and eradicate infected cells.
Dr. Edward Liu, an infectious disease specialist, underscores the significance of this research as a novel approach to suppressing HIV.
He notes the current reliance on drug combinations to manage the virus and highlights the potential of eliminating the virus within infected cells to prevent further spread and immune system damage.
Dr. Liu also points out the cost-effectiveness of this nanomedicine compared to conventional treatments.
Moving forward, Dr. Emmanuel Ho’s (the lead author) team is exploring avenues to optimize the technology and enhance its efficacy, while Dr. Liu emphasizes the necessity for further research to evaluate potential side effects and real-world effectiveness in preventing HIV infection.
Another study from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center (UNMC) has shown that gene-editing therapy targeting both HIV-1 and CCR5 effectively eradicates HIV infection.
Kamel Khalili, Ph.D., from the Lewis Katz School of Medicine, explains the rationale behind this approach, drawing on observations from reported HIV cures in human patients.
“In the few instances of HIV cures in humans, the patients underwent bone marrow transplantation for leukemia, and the donor cells that were used carried inactivating CCR5 mutations,” said Dr. Khalili.
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