ISIS Neutron and Muon Source Data Journal

Bio-mimetic guided design of novel helper lipids for gene therapy

Abstract: There has been a shift in the pharma industry towards development of medicines acting within the target cell, typically the in cytosol, thereby creating demand for advanced delivery vehicles that deliver their payload intracellularly. Generally, these vehicles are taken into the cell's endosome via a process known as endocytosis. In order for the therapeutic agent to exert its effect, the vehicle (and its payload) must escape the endosome into the cytosol. Currently only 2% of vehicles are released from the endosome into the cytosol. This study wishes to determine the detailed structure of lipid nanoparticles containing a novel bio-lipid, known as LPBA, that exhibits good transfection ability by disrupting the endosomal membrane using SANS in combination with contrast variation with a view to understanding how the lipid exerts its activity.

Principal Investigator: Professor Jayne Lawrence
Local Contact: Dr James Doutch
Local Contact: Dr Najet Mahmoudi
Experimenter: Dr Xianbing Dai
Experimenter: Dr Dave Barlow
Experimenter: Miss Yining Ma

DOI: 10.5286/ISIS.E.RB2510367

ISIS Experiment Number: RB2510367

Publisher: STFC ISIS Neutron and Muon Source

Data format: RAW/Nexus
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Data Citation

The recommended format for citing this dataset in a research publication is as:
[author], [date], [title], [publisher], [doi]

For Example:
Professor Jayne Lawrence et al; (2025): Bio-mimetic guided design of novel helper lipids for gene therapy, STFC ISIS Neutron and Muon Source, https://doi.org/10.5286/ISIS.E.RB2510367

Data is released under the CC-BY-4.0 license.