Author Archives: Karely Vega, MD

CRISPR-Cas3 System Restores Dystrophin Function in Stem Cells Derived from Patients with Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene.  On August 24th, 2023 in the journal Stem Cell Reports, researchers show how a dual CRISPR RNA method restored dystrophin protein function in induced pluripotent stem cells derived from DMD patients.  The approach worked by removing large sections of […]

Metabolite in Urine Predicts Diabetic Kidney Failure

Urine levels of adenine, a metabolite produced in the kidney, are predictive and a causative biomarker of looming progressive kidney failure in patients with diabetes, a finding that could lead to earlier diagnosis and intervention, researchers from The University of Texas Health Science Center at San Antonio (also called UT Health San Antonio) reported Aug. […]

Cells With an Ear for Music Release Insulin

Diabetes is a condition in which the body produces too little or no insulin. Diabetics thus depend on an external supply of this hormone via injection or pump.  Researchers led by Martin Fussenegger from the Department of Biosystems Science and Engineering at ETH Zurich in Basel want to make the lives of these people easier […]

Genetic Risk Factors for Heart Failure

In a new study co-led by investigators at the United States Department of Veterans Affairs and Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, a global team of scientists conducted one of the largest genetic association studies on heart failure to date.  Using genomic data from over 90,000 heart […]

New Approach to Nongenetic T-Cell-Based Immunotherapy

Immunotherapies for cancer aim to induce the immune system to combat cancer cells more effectively.  In the journal Angewandte Chemie, a Chinese research team has now described a new, modular strategy for T-cell-based immunotherapy that manages to work without complex genetic modifications.  Modulation of cell-cell communications through an ingenious regulatory circuit using various small, specially […]

New Antifungal Therapy for Fungal Meningitis

A team of University of Minnesota Medical School researchers successfully tested a new antifungal therapy to treat fungal meningitis.  The trial results were published in the peer-reviewed journal Clinical Infectious Diseases. The research team tested a new oral formulation of the antifungal medication amphotericin among people who had HIV and cryptococcal meningitis — a common […]

Evidence That Parkinson’s Starts in the Gut

The gut-first theory of Parkinson’s, originally proposed 20 years ago, started to intrigue Sulzer after his own research pointed toward the role of an autoimmune response in Parkinson’s. In Parkinson’s, a protein called alpha-synuclein becomes misfolded, accumulates inside neurons, and slowly poisons the cells.  Sulzer’s lab in collaboration with immunologists at the La Jolla Institute […]

Treating Obesity (And Brain) With Liraglutide

Obesity leads to altered energy metabolism and reduced insulin sensitivity of cells. The so-called “anti-obesity drugs” are increasingly used to treat obesity and have caused tremendous interest, especially in the USA.  Researchers at the Max Planck Institute for Metabolism Reseat reduced insulin sensitivity affects learning of sensory associations. A single dose of the anti-obesity drug […]

Cell Therapy Repairs Cornea Damage in Humans

A team led by researchers from Mass Eye and Ear, a member of Mass General Brigham, reports the results of a phase I trial of a revolutionary stem cell treatment called cultivated autologous limbal epithelial cell transplantation (CALEC), which was found to be safe and well-tolerated over the short term in four patients with significant […]

Simple Blood Test May Predict Future Heart Risk

A simple blood test may predict the risk of progressive heart and kidney disease in people with Type 2 diabetes and kidney disease, according to new research published today in the American Heart Association’s flagship journal Circulation. “Treatment with canagliflozin, a sodium glucose co-transporter 2 inhibitor, lowered biomarker levels and reduced the risk of hospitalization […]