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Gene Therapy AOP - Recent Educational Updates

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Unveiling molecular secrets: Analysis of stable lentiviral packaging cell lines enables identification of novel viral gene functions


Development of an AAV-RNAi strategy to silence the dominant variant <i>GNAO1</i> c.607G&gt;A linked to encephalopathy


Expression of anti-amyloid CARs in microglia promotes efficient and selective phagocytosis of Aβ1‒42


Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy?


Early life safety profiling of gene therapy for spinal muscular atrophy


Organoids – the future of pre-clinical development of AAV gene therapy for CNS disorders


Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application


CRISPR targeting of SNPs associated with age-related macular degeneration in ARPE-19 cells: a potential model for manipulating the complement system