A new CF treatment strategy involves small, nucleic acid molecules called oligonucleotides that can correct some of the gene defects that underlie CF but are not addressed by existing modulator therapies. The researchers used a new delivery method that overcomes traditional obstacles of getting oligonucleotides into lung cells.
The scientific world has been delving ever deeper into cancer, scourging for even the tiniest biomolecule that could amp up the cure for the deadly disease. A recent discovery in this regard is the gene LIM domain only 1, whose coded protein has a role in tumor formation. In a new article in Chinese Medical Journal, researchers have reviewed studies detailing molecular features of this gene for potential practical applications in cancer cure.
An international team of researchers have shed new light on the early stages of viral evolution.
Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.
A preclinical study led by scientists at Sanford Burnham Prebys has established that AAV8-TNAP-D10--a gene therapy that replaces a key enzyme found in bone--may be a safe and effective single-dose treatment for hypophosphatasia (HPP). The study, published in the Journal of Bone and Mineral Research and performed in a murine model of the disease, further supports advancing the therapy toward human clinical trials.
ETH Zurich researchers have developed a gene switch that can be operated with the green LED light emitted by commercial smartwatches. This revolutionary approach could be used to treat diabetes in the future.
Sickle cell disease leads to chronic pain, organ failure, and early death in patients worldwide. A team led by Broad Institute of MIT and Harvard and St. Jude Children's Research Hospital has demonstrated a gene editing approach that efficiently corrects the mutation underlying SCD in patient blood stem cells and in mice. This treatment rescued disease symptoms in animal models, enabling long-lasting production of healthy blood cells, and could inspire a therapeutic strategy for SCD.
Scientists have developed a gene drive with a built-in genetic barrier that is designed to keep the drive under control. The researchers engineered synthetic fly species that, upon release in sufficient numbers, act as gene drives that can spread locally and be reversed if desired.
In a study of 11 medical-mystery patients, an international team of researchers led by scientists at the National Institutes of Health and the Uniformed Services University (USU) discovered a new and unique form of amyotrophic lateral sclerosis (ALS). Unlike most cases of ALS, the disease began attacking these patients during childhood, worsened more slowly than usual, and was linked to a gene, called SPTLC1, that is part of the body's fat production system.
UC San Diego scientists have developed a genetics toolkit that helps pave the way to a gene drive designed to stop Culex mosquitoes from spreading disease. Much less studied than other genera, Culex mosquitoes spread devastating afflictions stemming from West Nile virus, Japanese encephalitis virus and the pathogen causing avian malaria.