Sickle-cell disease, or sickle-cell anaemia (or drepanocytosis), is a life-long blood disorder characterized by red blood cells that assume an abnormal, rigid, sickle shape. Sickling decreases the cells' flexibility and results in a risk of various complications.
Cardiovascular medicine, hematology and pulmonary medicine may soon have the first-ever therapies to correct poor tissue oxygenation, a key driver of disease in millions, including peripheral artery disease, sickle cell disease, heart failure, stroke, emphysema and many others.
Scientists at St. Jude Children's Research Hospital have demonstrated how drug makers can avoid two key problems: toxicity and resistance. The researchers made slight changes to a small molecule to reduce its metabolism and elimination by the cellular detoxification network regulated by the pregnane X receptor (PXR). This research provides a framework to develop solutions to the long-standing issue of how to evade detoxification networks using medicinal chemistry. The findings were published today in Proceedings of the National Academy of Sciences.
CTCF is a critical protein known to play various roles in key biological processes such as transcription.
CRISPR has made headlines in recent years for its potential to help patients with conditions as diverse as blindness and sickle cell disease. However, bacteria were already using CRISPR as an immune system to combat viruses long before humans adopted it to combat genetic disorders.
To celebrate National DNA Day 2022, we spoke to science communicator Professor Matthew Cobb.
CRISPR gene editing—a form of “molecular scissors”—was used by UNSW researchers to investigate how deletions in one section of the genome might impact the expression of surrounding genes.
Organ-on-a-chip technology has provided a push to discover new drugs for a variety of rare and ignored diseases for which current models either don't exist or lack precision. In particular, these platforms can include the cells of a patient, thus resulting in patient-specific discovery.
Scientists have created a high-throughput method to help explain and regulate gene expression for the treatment of diseases like beta-thalassemia and sickle cell disease.
The CRISPR-Chip technology was first introduced in 2019 by Dr. Kiana Aran, an Assistant Professor at Keck Graduate Institute (KGI).
Even a single letter variation in a single gene, acquired from both parents, could mean a lifetime of pain and anemia for 20 million individuals across the world.
Scientists have developed an affordable, downloadable app that scans for potential unintended mistakes when CRISPR is used to repair mutations that cause disease.
Researchers at the University of Colorado Boulder and the University of Colorado Anschutz Medical Campus have developed a new way to diagnose diseases of the blood like sickle cell disease with sensitivity and precision and in only one minute.