by University of York Landscape of somatic mutations in SCD. a, Dot-plot showing the number of mutations per HSPC for each patient plotted against the patient age at the time of sampling. SNV mutation burdens of individual HSPC colonies from before GT, with correction for coverage, are displayed per patient. Mean mutation burdens per individual are...
by Brigham and Women’s Hospital Credit: Pixabay/CC0 Public Domain The blood-brain barrier (BBB) is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain tissue, but it also keeps out potential treatment for diseases that affect the...
by St. Jude Children’s Research Hospital A schematic representation of the overall genomic safe harbor identification strategy. a Selection of common pMEIs from healthy individuals with AF > 0.1. b Removing pMEIs significantly associated with gene expression (FDR less than 0.1 in eQTL mapping). c Removing pMEIs showing spatial proximity with oncogenes, tumor suppressor genes,...
by Jim Fessenden, University of Massachusetts Medical School sup-tRNAs suppress the mIdua-W401X mutation in HEK293 cells. Credit: Nature (2022). DOI: 10.1038/s41586-022-04533-3 Gene therapy researchers at UMass Chan Medical School have shown the first evidence that a suppressor transfer RNA (tRNA) therapy can restore protein production up to six months after treatment in a mouse model of the...
Today, cell and gene therapies treat and could even cure terminal diseases like leukemia and spinal muscular atrophy, but unlocking their full potential is still a challenge for therapy developers. Both types of therapies rely on living cells comprised of hundreds of thousands of biomolecules to work in unison. And in order for therapy developers to meet...
by IOS Press Immunohistochemistry for alpha-synuclein showing positive staining (brown) of an intraneural Lewy-body in the Substantia nigra in Parkinson’s disease. Credit: Wikipedia After several decades of pre-clinical development, cell- and gene-based therapies for Parkinson’s disease (PD) are now actively being explored. In this special supplement to the Journal of Parkinson’s Disease, “Repairing the Parkinsonian Brain,” experts...
by IOS Press In clinical trials of adults with Huntington’s disease (HD) the Q-Suite Motor Assessment Tool (Q-Motor) has proven to be helpful to detect and quantitate subtle motor abnormalities. With the anticipated arrival of preventive gene therapies that will most likely be administered to young children known to be carriers of the HD mutation,...
