New Gene-Editing Technique Makes Strides Towards HIV Resistance

According to the Centers for Disease Control (CDC), 1,155,792 people in the United States have been diagnosed with acquired immunodeficiency syndrome (AIDS) caused by the human immunodeficiency virus (HIV). HIV targets T-cells via the CCR5 gene receptor which serves as a channel for the virus into cells. The virus then replicates inside the T-cells, eventually killing the host cells. This destruction of T-cells ultimately results in a highly susceptible immune system.While countless research projects aspire to develop a cure for the devastating virus, an approved cure has yet to be determined. However, hopes for finding a cure remain as vast strides towards a cure have been accomplished by some promising research.

Using a new gene-editing technique, researchers for the Harvard Stem Cell Institute (HSCI) at Massachusetts General (MGH) and Boston Children's (BCH) hospitals have created an effective technique for blocking HIV from invading and destroying its subject's immune system. The researchers effectively and precisely used CRISPR-Cas gene-editing technology to edit clinically relevant genes out of human hematopoietic stem cells and T-cells. The team was able to remove the CCR5 gene receptor out of of hematopoietic stem cells and demonstrate that these cells could differentiate into functional blood cells without the CCR5 gene. This outcome suggests that gene-edited stem cells could be delivered into HIV patients by bone marrow transplantation. The procedure would result in an HIV-resistant immune system. Dr. David Scadden, co-director of HSCI, stated that the new work is "a tremendous first step in editing out what makes human cells vulnerable to HIV."

The team identified areas of caution regarding the future of the new gene-editing therapy such as unexpected complications with the new therapy and the potential difficulty involved in treating people in the areas where HIV is most prevalent.The team also believes the new therapy will be ready for human safety trials within 5 years. The new therapy will undergo animal trials, and once they are completed, the team will apply for phase I human trials.

The more I read about the advances in gene-editing techniques, the more I am humbled at how far medical technology has come in such a short time period. I am excited to follow the development of this therapy through its trials. Advances such as this will encourage hope globally regarding the devastating virus.                                                                                                                                                                                                                                                                                                                                                                                                                                                                       
 Article: http://news.harvard.edu/gazette/story/2014/11/a-promising-strategy-against-hiv/
Related Article: http://www.nejm.org/doi/pdf/10.1056/NEJMoa0802905

Feline Immunodeficiency Virus Integrase Model Could Help Scientists Understand HIV

AIDS is not only a human disease, cats can get AIDS also. Both HIV (Human Immunodeficiency Virus) and FIV (Feline Immunodeficiency Virus) rely on one of the same proteins, called integrase. This protein is what inserts the virus' DNA into an infected cell's DNA. Researchers Akram Alian and Meytal Galilee at the Technion-Israel Institute of Technology have been investigating this protein and may have discovered a weak point in integrase that has potential to be targeted by drug designers in the future.

Akram Alian working in the lab on FIV integrase research.

The researchers have created a detailed, 3-D molecular map of integrase that could also help in the understanding of how the protein works in HIV. One particular point of interest is a specific amino acid that they have found to be critical in how the protein assembles itself from simpler building blocks; this would be useful in creating an anti-HIV drug. The researchers have also determined that changing just one amino acid converts the integrase in FIV from its complex form to a more simplified version.

From studying the model of FIV integrase in detail, researchers have concluded that simple and complex forms have nearly identical backbones, which could make it easier for scientists to study the simpler form in the laboratory while knowing their discoveries will apply to the more complex version in HIV as well.

Akram and Galilee hope to continue their research and next focus on how FIV could evolve over time. They think this will help them predict how emergent resistant viral variants may evolve to escape or become resistant to future drugs.

I initially chose this article because I have a cat and know they test/vaccinate kittens at the vet for feline AIDS. Once I actually read it though it was interesting beyond that. Studying integrase in the FIV seems to be very promising for advances in combating HIV. Advances like this is are important as AIDS and HIV have such a large impact on society and are still not fully understood.

Article: http://www.sciencedaily.com/releases/2014/09/140916101808.htm