Data from: Biodegradable acoustic targeting for ultrasound-supported gene therapy (BATUS) in glioblastoma
Data files
Jul 10, 2026 version files 170.57 MB
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flow_analysis_GL261_cells.zip
3.94 MB
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flow_analysis_U87_cells.zip
3.65 MB
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in_vivo__ivis_images-_survival.zip
56.83 MB
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README.md
5.52 KB
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Relase_study_siRNA_122324.pzfx
93.98 KB
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RT-PCR__results_on_GL261_cells_for_PDL1_silencing.pzfx
72.93 KB
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siRNA_BBB_organ_on_a_chip_permeability.pzfx
81.98 KB
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survival_graph_analysis.pzf
913.57 KB
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TEM_images.zip
104.78 MB
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tumor_growth-analysis.pzf
207.01 KB
Abstract
Glioblastoma (GBM) remains one of the most challenging brain malignancies due to the restrictive nature of the blood–brain barrier (BBB), which severely limits effective drug and gene delivery. To overcome this, we introduce Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS), a modular platform that enables safe, repeated, and targeted gene delivery to the brain. BATUS integrates an implantable, fully biodegradable glycine-based ultrasound (US) transducer, surgically placed via craniotomy for precise BBB opening, along with customizable peptide-targeted liposomal gene carriers. This system combines US-mediated transient BBB disruption with ligand-directed cellular targeting to enable efficient delivery across both vascular and cellular barriers. As a proof-of-concept, BATUS was evaluated using tLyp1-functionalized liposomes carrying PD-L1-targeting siRNA for GBM immunotherapy: in vitro evaluations confirmed efficient siRNA entrapment, stability, and specific cellular uptake, while in vivo studies in orthotopic GL261 GBM mouse models showed enhanced BBB permeability, significant PD-L1 silencing, reduced tumor growth, and prolonged survival. Crucially, BATUS exhibited a favorable safety profile, with no detectable local or systemic toxicity in animal models. By providing a modular framework where targeting ligands and genetic cargo are easily adaptable, BATUS is established as a strategy for precision gene delivery across the BBB, as demonstrated here for GBM.
Dataset DOI: 10.5061/dryad.p8cz8wb6h
Description of the data and file structure
The data were generated to evaluate the development and therapeutic performance of tLyp1-functionalized liposomal siRNA nanoparticles combined with biodegradable ultrasound-mediated blood–brain barrier opening for glioblastoma treatment. The dataset includes in vitro characterization, siRNA release, BBB-on-a-chip permeability, cellular uptake, gene silencing, in vivo tumor growth and survival analyses, IVIS imaging, flow cytometry, and transmission electron microscopy (TEM) data supporting the findings presented in the associated manuscript.
Files and variables
File: flow_analysis_U87_cells.zip
Description: Compressed folder containing raw flow cytometry data files and analysis files from U87 cell experiments evaluating cellular uptake of fluorescently labeled liposomal siRNA formulations.
File: tumor_growth-analysis.pzf
Description: GraphPad Prism project containing individual tumor growth measurements, tumor volume calculations, graphical presentation, and statistical analyses of the in vivo therapeutic efficacy study.
File: survival_graph_analysis.pzf
Description: GraphPad Prism project containing the raw survival data, Kaplan–Meier survival curves, and log-rank statistical analyses used to evaluate animal survival following different treatment groups.
File: RT-PCR__results_on_GL261_cells_for_PDL1_silencing.pzfx
Description: GraphPad Prism XML project containing raw qRT-PCR data, normalized gene expression values, and statistical analyses evaluating PD-L1 gene silencing in GL261 cells following treatment with siRNA-loaded liposomes.
File: siRNA_BBB_organ_on_a_chip_permeability.pzfx
Description: GraphPad Prism XML project containing raw fluorescence permeability measurements and statistical analyses from the BBB-on-a-chip experiments assessing siRNA transport across the blood–brain barrier model.
File: Relase_study_siRNA_122324.pzfx
Description: GraphPad Prism XML project containing the raw fluorescence measurements, data processing, and statistical analyses for the in vitro siRNA release study from the liposomal formulation over time.
File: in_vivo__ivis_images-_survival.zip
Description: Compressed folder containing raw IVIS fluorescence imaging data collected during the in vivo biodistribution and therapeutic studies, together with representative images associated with the survival experiments.
File: TEM_images.zip
Description: Compressed folder containing the original transmission electron microscopy (TEM) images used for characterization of the liposomal formulations and nanoparticle morphology.
File: flow_analysis_GL261_cells.zip
Description: Compressed folder containing raw flow cytometry data files and analysis files from GL261 cell experiments evaluating cellular uptake of fluorescently labeled liposomal siRNA formulations.
File: GEA_U87_V2_11212024.xml
Description: XML workspace containing the flow cytometry analysis performed on U87 glioblastoma cells to evaluate the cellular uptake of fluorescently labeled liposomal formulations. The file includes the gating strategy, fluorescence intensity analysis, and associated metadata.
File: GEA.xml
Description: XML workspace containing the flow cytometry analysis performed on GL261 glioblastoma cells to evaluate the cellular uptake of fluorescently labeled liposomal formulations. The file includes the gating strategy, fluorescence intensity analysis, and associated metadata.
File: raw_data_survival.zip
Description: Compressed folder containing the original IVIS Spectrum Living Image acquisition files collected during the in vivo therapeutic efficacy and survival studies. Each subfolder (e.g., GEA20250310102319, GEA20250318131140) corresponds to a single imaging session acquired on the indicated date and time. Each subfolder contains the original luminescent.TIF (bioluminescence image), photograph.TIF (photographic image), readbiasonly.TIF (camera background image), and the associated ClickInfo files containing acquisition parameters, imaging settings, and metadata generated by the Living Image software.
Code/software
The GraphPad Prism project files (.pzf and .pzfx) can be opened using GraphPad Prism (Version 10 or later). Flow cytometry data can be viewed and analyzed using FlowJo (Version 10 or later) or other software compatible with standard flow cytometry data formats. The TEM and IVIS image files are provided in standard image formats and can be opened using common image-viewing software such as ImageJ/Fiji or any standard image viewer. No custom scripts or source code were used to generate or analyze the data included in this repository.
Access information
Other publicly accessible locations of the data:
- None. The data are publicly available only through this Dryad repository.
Data were derived from the following sources:
- The data were generated by the authors through original in vitro and in vivo experiments, including cell culture studies, blood–brain barrier-on-a-chip experiments, flow cytometry, IVIS imaging, transmission electron microscopy (TEM), and orthotopic glioblastoma mouse studies. No external datasets were used.