Researchers have used gene therapy to prevent type 1 diabetes in mice, providing proof of principle that gene therapy can be used to treat autoimmunity. Moreover, in combination with low doses, an antibody targeting the costimulatory molecule CD3, the gene therapy was able to reverse the symptoms of type 1 diabetes in the animals.
When drug developers ponder how to get across the blood-brain barrier, it is more often within the context of how to get things into the brain. But new research suggests that in Alzheimer's disease, one of the issues is that amyloid-beta can't cross in the other direction and get out of the brain.
At a joint workshop of the FDA and the American Association for Cancer Research (AACR) last week, industry and regulators had a frank conversation about how the search for optimal dosing of cancer drugs needs to change.
Researchers at Boston Children's Hospital have used a novel type of screening to identify a compound that caused massive weight loss in mice with diet-induced obesity, causing them to lose 45 percent of their body weight.
By equipping bacteriophages with the gene-editing system CRISPR, researchers have been able to sensitize drug-resistant bacteria to antibiotics. And by combining that sensitization with enhanced resistance to a second separate phage, the team was able to forestall the development of resistance against the first phage – which left the bacteria vulnerable to antibiotics but did not kill them outright.
A genomics-based search for better drug targets for painkillers has turned up a candidate that is, in one sense, not new at all. That candidate is the nicotinic acetylcholine receptor, already the beneficiary of one failed round of drug development.
Scientists from Cold Spring Harbor Laboratories have adapted the CRISPR/Cas9 gene editing technique in a way that enabled them to comprehensively search for cancer drug targets, by targeting CRISPR to specific protein-coding domains rather than the 5-prime exons of genes.
The global public health community is rejoicing at news this week that the Ebola epidemic is over in Liberia, with no new cases reported in 42 days. And with only nine new cases reported overall last week, seven in Guinea and two in Sierra Leone, the current epidemic, after 18 months and more than 11,000 deaths, may be on the verge of finally being over.
In animal studies, researchers have tied levels of the G protein-coupled channel subunit, GIRK3, in a midbrain area known as the ventral tegmental area (VTA), to the propensity to binge drink.
Understanding how genomic variation plays out at the level of gene expression in different tissues is a fundamental scientific question, but also a prerequisite for truly harnessing genomic information for therapeutic purposes.
