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UrduA $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children’s Hospital of Philadelphia (CHOP). The research will focus specifically on developing therapies for urea cycle disorders, which impact roughly 1 in every 35,000 children.
Tag: Metabolic Diseases
Researchers design an innovative strategy to fight obesity through gene therapy
A scientific team from the University of Barcelona and the CIBERobn has designed a strategy to fight obesity and diabetes in mice through ex vivo gene therapy which consists of implanting cells that have been manipulated and transformed in order to treat a disease. This is the first study to apply the ex vivo gene therapy technique to generate and implant cells that express the CPT1AM protein, an enzyme that plays a decisive role in many metabolic diseases such as obesity.
Researchers discover rare form of premature-aging syndrome
UT Southwestern researchers have discovered a new form of progeria, a rare premature-aging syndrome, in a man from Malaysia and traced its cause to a novel gene mutation.
MERCY MEDICAL CENTER TO LAUNCH THE MARYLAND BARIATRIC CENTER
Noted bariatric specialist Kuldeep Singh, MD, FACS, MBA, FASMBS, has joined Mercy Medical Center to establish a new clinical program: The Maryland Bariatric Center at Mercy, scheduled to open in January 2020.
Subtle Mitochondrial DNA Mutations Alter Expression of Nuclear Genes, with Profound Clinical Effects
Subtle changes in the DNA of mitochondria, the tiny power plants in cells, can have profound consequences for a patient’s health. Research on biological mechanisms suggests that relatively minor changes in mitochondrial energy flow and metabolism could offer significant future benefits to patients with complex diseases.