Search the tables and observations reproduced from the product literature guide. Rows may represent measurements, recipes or experimental context; they are not all independent experiments.
Table 1
Formulation: Initial screening | Component order: LC-1 DCL0004 / DSPC DC57005 / Cholesterol DCD-041 / DMG-PEG2000 DC40169 | Molar ratio (mol%): 50:10:38.5:1.5 | Figure-reported N/P: 10 | Experimental use: Initial cell screening; not the optimized route-specific composition
Table 1
Formulation: Liver / lung optimized DOE-07 | Component order: LC-1 DCL0004 / DOPE DC33634 / Cholesterol DCD-041 / DMG-PEG2000 DC40169 | Molar ratio (mol%): 30:30:39.5:0.5 | Figure-reported N/P: 5 | Experimental use: Intravenous liver delivery OR intratracheal lung delivery
Table 1
Formulation: Brain optimized DOE-11 | Component order: LC-1 DCL0004 / DOPE DC33634 / Cholesterol DCD-041 / DMG-PEG2000 DC40169 | Molar ratio (mol%): 60:30:8:2 | Figure-reported N/P: 7.5 | Experimental use: Intrathecal brain delivery
Table 2
Parameter: Lipid / aqueous phases | Reported information: Lipids in ethanol; RNA in 10 mM citrate buffer, pH 4.0
Table 2
Parameter: Mixing ratio | Reported information: Aqueous:ethanol = 3:1 (v/v)
Table 2
Parameter: Mixing method | Reported information: Rapid pipette mixing for in vitro formulations; microfluidic mixing for in vivo formulations
Table 2
Parameter: Total flow rate | Reported information: Not reported in the supplied Methods
Table 2
Parameter: Buffer exchange | Reported information: Overnight dialysis at 4 °C into 1× DPBS; 10 kDa MWCO
Table 2
Parameter: Cas9 RNA ratio | Reported information: Cas9 mRNA:sgRNA = 1:1 in vitro; 3:1 in vivo (mass ratio)
Table 2
Parameter: ABE RNA ratio | Reported information: ABE mRNA:sgRNA = 1:1 (mass ratio), in vitro and in vivo
Table 2
Parameter: Apparent pKa | Reported information: Not located in the supplied main paper or SI
Table 2
Parameter: Particle characterization | Reported information: Diameter, PDI, zeta potential and encapsulation efficiency compared in Supplementary Fig. 54 (n = 3). Exact tabulated means are not supplied; no precise values are inferred from the plot.
Table 2
Parameter: Stability studies | Reported information: Diameter/PDI assessed over 14 days at 4 °C and in a serum challenge (Supplementary Figs. 66–67, n = 3). These experiments do not establish the storage lifetime of neat LC-1.
Table 3
Cell population: hepatocytes | Route: Intravenous | Reported dose: 1 mg/kg | LC-1: 79.1% | ALC-0315: 37.5% | LP-01: 19.2%
Table 3
Cell population: neurons | Route: Intrathecal | Reported dose: 0.1 mg/kg | LC-1: 48.3% | ALC-0315: 19.2% | LP-01: 16.3%
Table 3
Cell population: astrocytes | Route: Intrathecal | Reported dose: 0.1 mg/kg | LC-1: 45.3% | ALC-0315: 24.3% | LP-01: 14.5%
Table 3
Cell population: lung epithelial cells | Route: Intratracheal | Reported dose: 0.5 mg/kg | LC-1: 27.3% | ALC-0315: 15.4% | LP-01: 7.8%
Table 3
Cell population: lung endothelial cells | Route: Intratracheal | Reported dose: 0.5 mg/kg | LC-1: 21.7% | ALC-0315: 7.7% | LP-01: 7.7%
Table 4
Endpoint: Hepatic Pcsk9 A-to-G editing (Sanger/EditR) | LC-1: 79.5% | ALC-0315: 57.5% | LP-01: 41.5%
Table 4
Endpoint: Plasma PCSK9 reduction, author-reported | LC-1: 85.7% | ALC-0315: 55.0% | LP-01: 23.4%
Table 4
Endpoint: Plasma LDL reduction, author-reported | LC-1: 74.7% | ALC-0315: 35.1% | LP-01: 18.7%
Table 5
Route: Intravenous | Reported dose: 2 mg/kg | Composition: 30:30:39.5:0.5 | Readout: Restored Fluc expression; follow-up to 4 weeks
Table 5
Route: Intrathecal | Reported dose: 0.1 mg/kg | Composition: 60:30:8:2 | Readout: Restored Fluc expression; follow-up to 4 weeks
Table 5
Route: Intratracheal | Reported dose: 1 mg/kg | Composition: 30:30:39.5:0.5 | Readout: Restored Fluc expression; follow-up to 4 weeks
Table 6
Route: Intravenous | Reported dose: 0.25 mg/kg | Selected composition: Liver/lung optimized | Readout / n: 6-hour luciferase expression; n = 3
Table 6
Route: Intrathecal | Reported dose: 0.125 mg/kg | Selected composition: Brain optimized | Readout / n: 6-hour luciferase expression; n = 3
Table 6
Route: Intratracheal | Reported dose: 0.25 mg/kg | Selected composition: Liver/lung optimized | Readout / n: 6-hour luciferase expression; n = 3
Table 7
Condition: pH 7.4 | LC-1: 27.5% | ALC-0315: 7.2% | LP-01: 5.4%
Table 7
Condition: pH 5.5 | LC-1: 59.6% | ALC-0315: 40.8% | LP-01: 24.3%