by Physician’s Briefing Staff A new gene therapy for sickle cell disease has been deemed safe by a U.S. Food and Drug Administration advisory panel, paving the way for full approval by early December 2023. The FDA had already decided that the therapy, known as exagamglogene autotemcel (exa-cel), was effective. Developed by Vertex Pharmaceuticals of...
by Chinese Academy of Sciences Retrograde AAV selectively targets D1-MSNs and rescues parkinsonian symptoms with chemogenetic modulation. Credit: SIAT Researchers from the Shenzhen Institute of Advanced Technology (SIAT) of the Chinese Academy of Sciences (CAS) and their collaborators have developed a gene therapy strategy to selectively manipulate Parkinson’s disease-affected circuitry and attenuate the core motor symptoms...
by University of Surrey Flow-chart summarizing the 5-CSRTT process. During the 5-CSRTT, fish were required to swim toward one of five spatially distinct LEDs when illuminated. Approaches to the illuminated light were ‘correct’ and the proportion of correct trials was a measure of attention. Prior to illumination, there was a variable-time (mean 5-s) inter-trial interval, and...
Peer-Reviewed Publication CENTRO NACIONAL DE INVESTIGACIONES ONCOLÓGICAS (CNIO) FROM LEFT TO RIGHT: IVÁN PLAZA-MENACHO, SENIOR AUTHOR, AND FIRST AUTHORS JULIA CONTRERAS AND HIPÓLITO NICOLÁS CUESTA HERNÁNDEZ.view more CREDIT: ESTHER SÁNCHEZ / CNIOIn the late 1970s, the relationship between the c-Src gene and cancer was discovered. The first oncogene was identified.Since then, c-Src has been found...
by Olivia Dimmer, Northwestern University Multiplex immunofluorescence staining and imaging using the COMET system platform on a human meningioma patient sample, showing SSTR2+ tumor cells (in red), expressing the hedgehog pathway activation marker sonic hedgehog (SHH, in cyan blue). Credit: Hinda NajemScientists have uncovered a genetic explanation for one subset of common brain tumors, according to...
by deCODE genetics Credit: Pixabay/CC0 Public DomainA large international study led by deCODE Genetics on the genetics of migraine provides novel insights into the biology of migraine enabling detection of rare variants protecting against migraine, opening an avenue for potential development of novel drug targets. In a study published in Nature Genetics a group of...
by Catherine Caruso, Harvard Medical School Credit: Pixabay/CC0 Public DomainWhen neurobiologist David Corey showed up at a rare disease conference in 2017, he had no idea that he would enter a race against time to develop a treatment for it. The conference was for Usher syndrome type 1F. Patients with this condition have a gene mutation...
byEric W. Dolan October 22, 2023 In a groundbreaking discovery published in the journal Neuron, scientists have challenged a long-held belief about the origins of Parkinson’s disease, shedding new light on this debilitating neurological condition. Their research suggests that the malfunctioning of synapses, the tiny gaps that allow neurons to communicate with each other, might...
by Cell Press This photo shows green dystrophin protein restored by dual-Cas3 in skeletal muscle derived from DMD patient iPSCs. Credit: Akitsu HottaDuchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. On August 24 in the journal Stem Cell Reports, researchers show how a dual CRISPR RNA method...
(Adobe Stock) Scientists have discovered two genes that may trigger Raynaud’s phenomenon, a condition that can cause fingers and toes to go cold and numb because of the constriction of tiny blood vessels under the skin. “We identify two distinct genes that point to two distinct mechanisms,” lead researcher Maik Pietzner, chair in health data...
