Clustered regularly interspaced short palindromic repeats (CRISPR) and a CRISPR associated protein (Cas9) comprise a system adapted from bacterial DNA known as CRISPR/Cas9. The CRISPR/Cas9 system act as an adaptive immune system allowing bacteria to recognize foreign DNA and cut it, destroying an invading virus or pathogen. CRISPR/Cas9 has been adapted to other organisms to make specific cuts in their genomic DNA.
CRISPR/Cas9 technology holds the potential to “edit” a patient’s genome, restoring genes with disease-causing mutations to their normal healthy sequence, deleting disease-causing genes, or improving genes … Read more
Abicipar is a DARPin-based anti-angiogenic drug for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). Wet AMD is a leading cause of blindness in the developed world and DME is a leading cause of blindness in young adults in developed countries.
Abicipar is an antagonist of Vascular Endothelial Growth Factor A (VEGF-A) that inhibits all relevant subtypes of VEGF-A with very high potency. The combination of small size, high potency and long intra-vitreal half-life offers the potential for less frequent injections (compared to Lucentis, the current
Studying the muscle stem cells in boys suffering fromDuchenne muscular dystrophy, Stanford scientist Helen Blau found that their telomeres–the protective caps at the end of chromosomes–were unusually short. Now, in a new study, Blau and a small group of colleagues say that they have developed a method to use modified messenger RNA to lengthen telomeres, opening a pathway to countering the effects of aging in cells–a process that might apply to a range of diseases from DMD to diabetes and heart disease.
Telomeres naturally shorten every time a … Read more
The FDA is evaluating proposals for new indications for at least 17 oncology medicines, according to Friends of Cancer Research. The list of therapies includes novel agents and existing drugs whose uses could be expanded if the agency approves the applications.
The breakthrough therapy program is designed to streamline drug development for therapies to treat serious and life-threatening illnesses. The FDA grants the status based on whether a therapy would provide a substantial improvement over existing options based on preliminary clinical evidence.
The Friends advocacy group helped promote the concept … Read more
Diabetes Mellitus (DM) is a metabolic disorder, cause usually by a combination of hereditary and environmental factors, and resulting in abnormally high blood sugar levels (hyperglycemia). DM results following impaired insulin production by the pancreatic islet cells. The most common types of the disease are type-1 DM (T1DM) and type-2 DM (T2DM).
In T1DM, the onset of the disease follow an autoimmune attack of beta cells thus causing a severe reduction in beta cell mass. In T2DM, the pathogenesis involves insulin resistance, insulin deficiency and enhanced gluconeogenesis, while late progression … Read more