New Findings Opens Path For Alternative Treatments for Cystic Fibrosis
A new study completed by scientists at Rockefeller University uncovered the functions of a drug often used to target the mutated protein in people with cystic fibrosis. While the drugs, known as potentiators, can alleviate symptoms in some patients, much has remained unknown on how the drugs function. The recent findings characterize the interaction between potentiators and the protein they target and demonstrate that two distinct compounds act on the same protein region. In the report, researcher and professor Jue Chen, wrote: "These compounds are developed by two different companies and have very different chemical properties. But they manage to make their way to the same site. That tells us that this is a very sensitive, very important region of the protein." The researchers hope to use this knowledge to build compounds which directly target the area. Professor Chen noted: "We put our original data online and welcome anyone to use it. Because if more researchers use it, more treatment options will become available, prices will drop, and more people will be helped."
Researchers Eliminate Virus Responsible for AID From Genomes of Living Animals
For the first time, researchers have been able to eliminate the virus responsible for HIV-1, replication component HIV-1 DNA, from the genome of living animals, marking a pivotal move towards the development of a potential cure for human HIV. Reported in Nature Communications, the research demonstrated that treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals. The research was completed by a diverse team which included virologists, immunologists, molecular biologist, pharmacologist, and pharmaceutical experts. In a statement, researcher Kamel Khalili, PhD, noted: “The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection. We now have a clear path to move ahead to trials in non-human primates and possibly clinical trials in human patients within the year."
New Study Offers Insight On How To Prevent Brain Tissue Inflammation
New research from scientists at the Massachusetts General Hospital offers insight on how to prevent inflammation of brain tissue which contributes to Alzheimer’s disease (AD). The new findings may potentially serve to the development of new treatment options for the disease. In analyzing genes associated with neuroinflammation in AD, the researcher’s discovered the first gene associated with neuroinflammation, CD33 in 2008. In their latest research, the scientists sought to understand what would occur when critically important genes are silenced individually and simultaneously. The researchers found that when mice had CD33 turned off, they had reduced levels of amyloid plaque in their brains and performed better than other AD mice on test of learning and memory. When both CD33 and TREM2 (another associated gene) were silenced, the brain and behavior benefits disappeared. One researcher, neuroscientist Rudolph E. Tanzi, PhD, explained: “That tells us that TREM2 is working downstream of CD33 to control neuroinflammation. We are increasingly realizing that to help Alzheimer's patients, it is most critical to stop the massive brain nerve cell death that is caused by neuroinflammation. We now see that the CD33 and TREM2 genes are the best drug targets for achieving this goal."