| |  A new generation of CRISPR technology developed at UNSW Sydney offers a safer path to treating genetic diseases like Sickle Cell, while also proving beyond doubt that chemical tags on DNA - often thought to be little more than genetic cobwebs - actively silence genes. | |
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| |  Much the way the caps on the ends of a shoelace prevent it from fraying, telomeres - regions of repetitive DNA sequences and a protein structure - protect the tips of chromosomes from damage. | |
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| |  A computational approach by St. Jude Children's Research Hospital scientists promises to make designing T cell-based immunotherapies that target two cancer-related antigens at the same time far easier and faster. | |
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| |  Neovascular age-related macular degeneration (nAMD) is a progressive eye disease characterized by choroidal neovascularization and subretinal hemorrhage and exudation, leading to vision impairment. Current first-line treatment for nAMD are anti-VEGF agents, which has shown promising therapeutics outcomes. | |
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| |  Patients in a Phase I/II clinical trial conducted by UMass Chan Medical School of a dual vector gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases, exhibited a biochemical correction of the disease with minimal adverse reactions. | |
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| |  Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author of a new paper published in Signal Transduction and Targeted Therapy, "AAV for gene therapy drives a nephrotoxic response via NFκB in kidney organoids." | |
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| |  Traumatic injuries of the central nervous system (CNS)-such as traumatic brain injury (TBI) and traumatic spinal cord injury (SCI)-are characterized by oxidative damage and neuroinflammation. | |
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| |  With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine. But until scientists can deliver its gene-editing machinery safely and efficiently into relevant cells and tissues, that promise will remain out of reach. | |
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| |  A research team headed by the University of Zurich has developed a powerful new method to precisely edit DNA by combining cutting-edge genetic engineering with artificial intelligence. | |
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| |  In the past decade there has been significant interest in studying the expression of our genetic code down to the level of single cells, to identify the functions and activities of any cell through the course of health or disease. | |
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| |  With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine. | |
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