OCT 26, 2019 9:37 AM PDT

Gates Foundation and NIH Pledge $200 Million for Gene Therapies in Africa

WRITTEN BY: Annie Lennon

Over the next four years, the National Institutes of Health and the Bill and Melinda Gates Foundation plan to each invest $100 million towards the development of gene therapy-based cures for sickle cell disease and HIV. In particular, they are focusing on making these treatments available in sub-Saharan Africa, where they are needed most. 

According to NIH Director Dr. Francis Collins, “This unprecedented collaboration focuses from the get-go on access, scalability and affordability of advanced gene-based strategies for sickle cell disease and HIV to make sure everybody, everywhere has the opportunity to be cured, not just those in high-income countries...We aim to go big or go home (Ducharme: 2019).”

Gene therapy research has made some significant advances in recent years. Already, it is being used to treat several diseases, from inherited blindness to neuromuscular disease and leukemia. And now, both animal studies and some clinical trials have suggested that gene therapy technology may be able to treat two highly prevalent diseases in Africa: sickle cell disease and HIV (Cohen: 2019). 

To treat sickle cell disease, the collaboration aims to use in vivo gene therapy to deliver a therapeutic gene or gene-editing tools directly into the body via vectors, such as inactive viruses of nanoparticles. According to Collins, the procedure should resemble a simple blood transfusion. 

Although studies are already underway investigating ways that viral vectors can be used to deliver healthy genes to various tissues in the body, so far, in vivo gene therapy has only been used to modify blood stem cells in animal models of certain diseases. Thus, the collaboration aims to build upon this research and build ways for this technology to work in people too. 

As for HIV, the collaboration hopes to quicken the development of direct injections of gene editing components to target the CCR5 gene in white blood cells. With two people already having been cured of the disease via this treatment, in crippling the CCR5 gene, HIV is unable to enter new host cells and thus has been shown to naturally peter out. According to Steven Deeks, a leading HIV cure researcher at the University of California, San Francisco, “The potential beauty of in vivo gene editing is that it might be given ultimately as a single shot, curing everyone in a scalable manner (Ibid.).”


Sources 

 

Ducharme, Jamie: Time 

Cohen, Jon et al.: Science

About the Author
  • Science writer with keen interests in technology and behavioral biology. Her current focus is on the interplay between these fields to create meaningful interactions, applications and environments.
You May Also Like
APR 09, 2020
Genetics & Genomics
APR 09, 2020
As the Knee Evolved, So Did Arthritis Risk
As humans evolved, they began to walk upright, which has helped propel our species forward, literally and figuratively.
APR 13, 2020
Microbiology
APR 13, 2020
Even Bacteria Align With the Daily Cycle of Day and Night
The majority of living organisms on Earth have adapted in some way to the daily cycle of night and day.
APR 21, 2020
Cell & Molecular Biology
APR 21, 2020
Virtual Cell Provides a Close Look at Gene Expression During Development
Living organisms start out as one cell, and its genetic programs allow it to divide many times over, to give rise to the ...
APR 28, 2020
Genetics & Genomics
APR 28, 2020
Evolution Observed in Fish in a Single Generation
In a five year study, scientists have now shown that stickleback fish were able to alter some of their traits within a s ...
MAY 09, 2020
Clinical & Molecular DX
MAY 09, 2020
Rapid, Efficient COVID-19 Diagnostic Method Relies on Magnetic Beads
The pandemic virus SARS-CoV-2 has spread widely, and a lack of testing has been a serious problem.
MAY 24, 2020
Genetics & Genomics
MAY 24, 2020
How CRISPR Can Aid in Wildlife Conservation
Since it was developed, researchers have modified and applied the CRISPR gene editing technology in many different ways.
Loading Comments...