Designs

Canonical constructs that combine parts into validated gene therapy systems. Bridging literature to manufacturable products.

NameValidated In
(pro)-(ins)-WPRE-SV40pA —
(ins) —
(pro)-(ins) —
(pro)-(ins)-P2A-EGFP-WPRE-SV40pA —
(pro)-OpenCRISPR-1-SV40pA + 2*{U6-(gRNA)}-CAG-EGFP-KASH-bGHpA HEK293 cells (OpenCRISPR-1 characterization); dual-gRNA companion architecture validated in mammalian cells
(pro)-OpenCRISPR-1-SV40pA + 2*{U6-(gRNA)}-CAG-mCherry-bGHpA HEK293 cells (OpenCRISPR-1 characterization); dual-gRNA companion architecture validated in mammalian cells
(pro)-OpenCRISPR-1-SV40pA + U6-(gRNA)-CAG-EGFP-KASH-bGHpA HEK293 cells (OpenCRISPR-1 characterization); companion vector architecture validated in mouse neurons in vivo
(pro)-OpenCRISPR-1-SV40pA + U6-(gRNA)-CAG-mCherry-bGHpA HEK293 cells (OpenCRISPR-1 characterization); companion vector architecture validated in mammalian cells
(pro)-OpenCRISPR-1-SV40pA + U6-(gRNA)-(ins) HEK293 cells (OpenCRISPR-1 characterization)
(pro)-SpCas9-SV40pA + 2*{U6-(gRNA)}-CAG-EGFP-KASH-bGHpA —
(pro)-SpCas9-SV40pA + 2*{U6-(gRNA)}-CAG-mCherry-bGHpA —
(pro)-SpCas9-SV40pA + 2*{U6-(gRNA)}-CAG-NLS-EGFP-bGHpA —
(pro)-SpCas9-SV40pA + 3*{U6-(gRNA)}-CAG-EGFP-KASH-bGHpA —
(pro)-SpCas9-SV40pA + 3*{U6-(gRNA)}-CAG-mCherry-bGHpA —
(pro)-SpCas9-SV40pA + 3*{U6-(gRNA)}-hSyn-mCherry-KASH-bGHpA —
(pro)-SpCas9-SV40pA + U6-(gRNA)-CAG-EGFP-KASH-bGHpA —
(pro)-SpCas9-SV40pA + U6-(gRNA)-CAG-mCherry-bGHpA —
(pro)-SpCas9-SV40pA + U6-(gRNA)-hSyn-mCherry-bGHpA —
(pro)-SpCas9-SV40pA + U6-(gRNA)-(ins) —
{U6-(gRNA)}*2-(ins) —
{U6-(gRNA)}*3-hSyn-mCherry-KASH-hGHpA Neuro2a cells; mouse suprachiasmatic nucleus (SCN) in vivo, AAV-DJ serotype; organotypic SCN slice cultures
U6-(gRNA)-CMV-Cre-P2A-mCherry-pA —
U6-(gRNA)-CMV-EGFP-WPRE-pA —
U6-(gRNA)-hSyn-Cre-P2A-EGFP-KASH-hGHpA Mouse neurons in vivo (cortex, hippocampus); Cas9 knock-in mouse lines
U6-(gRNA)-hSyn-EGFP-KASH-hGHpA Mouse neurons in vivo; Neuro2a cells
U6-(gRNA)-(ins) —
U6-(gRNA)-(pro)-ABE-NT-bGHpA + EFS-ABE-CT-bGHpA —
U6-(gRNA)-(pro)-ABE7.10-bGHpA —
U6-(gRNA)-(pro)-ABE8e-bGHpA —
U6-(gRNA)-(pro)-AncBE4max-bGHpA —
U6-(gRNA)-(pro)-BE3-bGHpA —
U6-(gRNA)-(pro)-BE4max-bGHpA —
U6-(gRNA)-(pro)-CBE-NT-bGHpA + EFS-CBE-CT-bGHpA —
U6-(gRNA)-(pro)-CGBE-bGHpA —
U6-(gRNA)-(pro)-CjABE8e-bGHpA —
U6-(gRNA)-(pro)-Nme2ABE8e-bGHpA —
U6-(gRNA)-(pro)-SaABE8e-bGHpA —
U6-(gRNA)-(pro)-SaABE8e-V106W-bGHpA —
U6-(gRNA)-(pro)-SaBE39max-NT-bGHpA + EFS-SaBE39max-CT-bGHpA —
U6-(gRNA)-(pro)-SaBE39max-NT-bGHpA + EFS-SaBE39max-KKH-CT-bGHpA —
U6-(gRNA)-(pro)-SaCBE-NT-bGHpA + EFS-SaCBE-CT-bGHpA —
U6-(gRNA)-(pro)-SaCBE-NT-bGHpA + EFS-SaCBE-KKH-CT-bGHpA —
U6-(gRNA)-(pro)-SaKKH-ABE8e-bGHpA —
U6-(gRNA)-(pro)-SauriABE8e-bGHpA —
U6-(gRNA)-(pro)-Target-AID-bGHpA —
U6-(SaCas9gRNA)-EFS-EGFP-KASH-hGHpA 293FT cells; mouse basal lateral amygdala neurons (in vivo, AAV-DJ serotype)
U6-(gRNA)-CMV-ZsGreen-pA Mouse neurons