by Children’s Medical Research Institute Marti Cabanes-Creus in the Translational Vectorology lab at Children’s Medical Research Institute. A new publication by scientists from Children’s Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the liver. The paper titled,...
UNIVERSITY OF PENNSYLVANIA SCHOOL OF MEDICINE PHILADELPHIA — Penn Medicine researchers have solved a decades old mystery around a key molecule fueling the power plant of cells that could be exploited to find new ways to treat diseases, from neurodegenerative disorders to cancer. Reporting in a new study published today in Nature, researchers from the Department...
by University of Helsinki A new model of ANGPT2-mediated TIE2 and TIE1 clustering and activation at atomic resolution. Credit: VM Leppanen et al., Sci. Transl. Med. 12, eaax8013 (2020) The Human Molecular Genetics laboratory of the de Duve Institute (UCLouvain), headed by Professor Miikka Vikkula, recently identified mutations in a novel gene, ANGPT2, responsible for primary lymphedema....
by Deborah Kotz, University of Maryland School of Medicine A surface view of the organ of hearing (cochlea) from a mouse, using confocal microscopy. The sensory cells are named hair cells because of their apical projections (stereocilia) which move from stimulation by sound. Credit: University of Maryland School of Medicine Researchers at the University of Maryland...
INDIANA UNIVERSITY SCHOOL OF MEDICINE IMAGE: SUTHAT LIANGPUNSAKUL, MD INDIANAPOLIS–Researchers at Indiana University School of Medicine are learning more about how a person’s genes play a role in the possibility they’ll suffer from alcoholic cirrhosis with the discovery of a gene that could make the disease less likely. Alcoholic cirrhosis can happen after years of...
NORTHWESTERN UNIVERSITY CHICAGO — The locus of male sexual desire has been uncovered in specific regions of brain tissue where a key gene named aromatase is present, reports a new Northwestern Medicine study in mice. The gene regulates sexual behavior in men, and thus can be targeted by drugs to either increase its function for...
by Bob Shepard, University of Alabama at Birmingham The neurons in this image are stained blue, indicating the presence of the BIN1 protein. Points of direct interaction between BIN1 and calcium channels are in purple. Credit: University of Alabama at Birmingham Researchers at the University of Alabama at Birmingham are on the track of a...
ASSOCIATE PROFESSOR OF PATHOLOGY, MEMBER OF THE PITTSBURGH LIVER RESEARCH CENTER AND THE MCGOWAN INSTITUTE FOR REGENERATIVE MEDICINE view more CREDIT: UNIVERSITY OF PITTSBURGH PITTSBURGH, Sept. 3, 2020 – Gene therapy generally relies on viruses, such as adeno-associated virus (AAV), to deliver genes into a cell. In the case of CRISPR-based gene therapies, molecular scissors...
UMEA UNIVERSITY ILLUSTRATION OF HOW THE PROTEIN SMAD7 ENHANCES THE DEVELOPMENT OF TUMORS BY REGULATING THE GENE EXPRESSION OF HDAC6 AND C-JUN. view more CREDIT: KEMAL AVICAN An increased awareness on a molecular level of what mechanisms prostate cancer cells use to become mobile and start spreading may in the long run provide new opportunities...
MARY ANN LIEBERT, INC./GENETIC ENGINEERING NEWS JOURNAL IN THE FIELD AND PROVIDES ALL-INCLUSIVE ACCESS TO THE CRITICAL PILLARS OF HUMAN GENE THERAPY: RESEARCH, METHODS, AND CLINICAL APPLICATIONS New Rochelle, NY, September 2, 2020—A meta-analysis of non-human primate (NHP) studies showed that adeno-associated virus (AAV) gene therapy often caused dorsal root ganglion (DRG) pathology. There were...
