Table 2
Component: 5A2-SC8 | Molecular weight: 1841.76 | Reported molar entry: 19 | Calculated mass: Not calculated
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.
Component: 5A2-SC8 | Molecular weight: 1841.76 | Reported molar entry: 19 | Calculated mass: Not calculated
Component: DOPE | Molecular weight: 744.03 | Reported molar entry: 19 | Calculated mass: Not calculated
Component: Cholesterol | Molecular weight: 386.65 | Reported molar entry: 38 | Calculated mass: Not calculated
Component: DMG-PEG2000 | Molecular weight: 2507 | Reported molar entry: 4 | Calculated mass: Not calculated
Component: Palmitic acid hydrazide (C6) | Molecular weight: Not confirmed | Reported molar entry: 20 | Calculated mass: Not calculated
Parameter: Cargo-specific loading | Reported procedure / limitation: Earlier Methods extraction: total lipid:RNA mass ratios 40:1 (luciferase), 20:1 (Cre), and 10:1 (Cas9 / base-editor cargo). | Source: 2024 study; these are not N/P ratios
Parameter: Covalent fifth component | Reported procedure / limitation: C6 is palmitic acid hydrazide; AA11 and SA-NHS are separate tested covalent lipids. Do not label all five-component particles as the same recipe. | Source: 2024 SI Results and Discussion
Parameter: Preparation gaps | Reported procedure / limitation: Exact phase ratio, buffer, mixing and purification parameters require checking the full 2024 Methods before reproducing a batch. | Source: Not inferred from the SI or the four-lipid ratio
Parameter: Older siRNA platform | Reported procedure / limitation: The 2016 siRNA work uses a different formulation context. Its lipid recipe and FVII knockdown results are not transferred to the 2024 bone-marrow system. | Source: 2016 article versus 2024 article/SI
Measurement / batch: Base composition | Reported result: 15:15:30:3 is a molar ratio; normalized percentages are calculated only for the mass table | Evidence context: Sum = 63
Measurement / batch: Five-component identity | Reported result: 20 mol% covalent lipid is a formulation design variable | Evidence context: 2024 SI Fig. 11
Measurement / batch: Size / encapsulation | Reported result: No single batch value is assigned across Luc, Cre, Cas9 and base-editor LNPs | Evidence context: Cargo and fifth-component identity must remain linked
Experiment and dose: Covalent-lipid mechanism control SA-NHS formulation versus hydrolyzed stearic acid or an unreactive headgroup analog | Readout and comparator: Readout: Luciferase organ-imaging assay Comparator: Matched negative-control formulations | Result and source: Hydrolyzed and unreactive comparators did not confer the same bone-marrow reporter delivery. The reactive component, rather than 5A2-SC8 alone, contributes to tropism. 2024 SI Fig. 2; SI Results
Experiment and dose: Ionizable-lipid comparison 20 mol% AA11 in 5A2-SC8-, SM-102- or ALC-0315-based LNP; C57BL/6 IV reporter experiment | Readout and comparator: Readout: 6 h Comparator: SM-102 and ALC-0315 study formulations | Result and source: Bone-marrow homing was retained across carrier classes; the 5A2-SC8 formulation gave stronger delivery in this comparison and was used subsequently. 2024 SI Fig. 4; dose not transcribed here
Experiment and dose: Covalent-lipid fraction AA11 at 10%, 20% or 30%; Cre mRNA in reporter mice | Readout and comparator: Readout: SI cell-transfection endpoint Comparator: 10% and 20% AA11 | Result and source: Raising AA11 from 10% to 20% did not improve all populations; 30% markedly reduced delivery, particularly in stem/progenitor cells. 2024 SI Fig. 11
Experiment and dose: Formulation storage C6-mDLNP with Cre mRNA; stored for 3 days before IV administration | Readout and comparator: Readout: After the study reporter-expression interval Comparator: Fresh particles; room temperature / −20 °C storage | Result and source: 4 °C storage maintained measured cell transfection; room temperature and −20 °C reduced activity. Particle-size/PDI results supported this trend. 2024 SI Figs. 12–13
Experiment and dose: Ex vivo sickle-cell editing workflow Townes-mouse CD117+ cells; 10 pg RNA/cell; ABE8e_NRCH mRNA:sgHBB = 2:1 by mass | Readout and comparator: Readout: 14-day erythroid differentiation; separate 18 h hypoxia assay Comparator: Study differentiation controls | Result and source: Editing and erythrocyte phenotypes were evaluated in an ex vivo workflow. No in vivo cure percentage is inferred from these methods. 2024 SI Methods, printed p.3
Experiment and dose: Acute tolerability markers C57BL/6J mice; C6-LNP luciferase mRNA 0.6 mg/kg IV | Readout and comparator: Readout: 24 h and day 7 Comparator: Study controls | Result and source: Some bone/marrow and complement-related markers changed at 24 h; measured markers returned toward baseline at day 7. This is not evidence of no acute response. 2024 SI Figs. 21–23