Engineering the mammalian genome

A.Mallamaci, 22 hours


  • Goals of manipulations, gateways to the genome, molecular machineries

  • Germline transgenesis in zygote: classical and lentiviral

  • Gene trapping: NHEJ- and LV- assisted in ESCs, PiggyBac trapping in zygotes

  • Site-specific recombinases and their use in conditional mutagenesis

  • Conditional transgenesis by ligand-regulatable transcription

  • Gene editing, classical in ESCs

  • Engineered endonucleases (ZFNs, TALENs, CRISPR) and their use in gene editing

  • DNA base editing

  • Modulation of endogenous gene expression by programmable epigenetic effectors, programmable transcription factors, small activating RNAs and RNAi effectors

  • Modulation of endogenous gene expression by programmable translational transactivators (SineUps) and polypeptide Small Molecule-Assisted Shutoff (SMASh)