OSCR

Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS) in Glioblastoma.

Overview

Authors: Gulsah Erel‐Akbaba1,2, Jinyoung Park3, Achal Duhoon4, Cao Thuy Giang Nguyen1, Nitu Bhaskar5, Sumanta Kumar Karan5, Hasan Akbaba1,6,7, Parbeen Singh1, Thi Bao Tram Tran3, Yuhui Zhu1, Zhiming Li3, I'jaaz Muhammad3, Xiaojun Zhang3, Hoang Quan Truong1, A. Daniel Davidar8, Elizabeth Schmitzer9, Vikas N. Vattipally8, Angelica F. Lopez8,9, Patrick Kramer8, Victor M. Quiroz9
and 6 other authorsBen Bykov8, Claire Hao8, Amir Manbachi8,9, Nicholas Theodore8, Joshua C. Doloff9,10,11, Thanh D. Nguyen1,3,4
  1. Polymer Program Institute of Materials Science University of Connecticut Storrs Connecticut USA
  2. Department of Pharmaceutical Biotechnology Faculty of Pharmacy Izmir Katip Celebi University Izmir Turkey
  3. Department of Biomedical Engineering University of Connecticut Storrs Connecticut USA
  4. Department of Mechanical Engineering University of Connecticut Storrs Connecticut USA
  5. Center for Clean Energy Engineering University of Connecticut Storrs CT USA
  6. Ege University Vaccine Development Application and Research Center Izmir Turkey
  7. Department of Pharmaceutical Biotechnology Faculty of Pharmacy Ege University Izmir Turkey
  8. Department of Neurosurgery Johns Hopkins School of Medicine Baltimore Maryland USA
  9. Department of Biomedical Engineering Johns Hopkins School of Medicine Baltimore Maryland USA
  10. Department of Materials Science and Engineering Johns Hopkins University Baltimore Maryland USA
  11. Department of Oncology Division of Cancer Immunology Sidney‐Kimmel Comprehensive Cancer Center Bloomberg∼Kimmel Institute for Cancer Immunotherapy Johns Hopkins University Baltimore Maryland USA
Institutions: University of Connecticut (United States); Izmir Kâtip Çelebi University (Türkiye); Ege University (Türkiye); Johns Hopkins University (United States); Johns Hopkins Medicine (United States); Sidney Kimmel Comprehensive Cancer Center (United States)
Journal: Advanced science (Weinheim, Baden-Wurttemberg, Germany), volume 13, issue 53, article e76336
Dates: received 17 April 2026; accepted 17 June 2026; published online 29 June 2026; in print September 2026
Type: Research article · Language: English
License: CC BY
Identifiers: DOI 10.1002/advs.76336 · PMID 42371791 · PMCID PMC13336871 · OpenAlex W7166588621
Open access: gold, a free copy (OpenAlex)
Status: data only
Categories: other (modality), human (organism), mouse (organism), other condition (population), cellular / molecular (subfield)
Keywords: biodegradable ultrasound transducer, blood–brain barrier opening, glioblastoma immunotherapy, piezoelectric biomaterials, ultrasound‐mediated gene delivery
MeSH: Brain Neoplasms*, Gene Transfer Techniques*, Genetic Therapy*, Glioblastoma*, Animals, Blood-Brain Barrier, Cell Line, Tumor, Disease Models, Animal, Gene Therapy Agents, Humans, Mice (* major topic)
Topic: Ultrasound and Hyperthermia Applications (Biomedical Engineering, Engineering), according to OpenAlex
Funding: NIH/NINDS (R01NS131310, R21NS116095); NIH/NIBIB (R01EB036924); NIH/NIAMS (R21AR078744)
Citations: not cited yet (Europe PMC); 74 references in the paper

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.

Reproduced under the paper's license (CC BY), from the paper cited above.

Code

The paper links to its data, not to its authors' code: see the Data section.

Tracing map

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Data

Datasets cited

Data Availability Statement

The main data supporting the results of this study are available within the paper and its Supplementary Information. The raw data is also published in the DRYAD. The remaining raw and analyzed datasets from the study are available for research purposes upon reasonable request from the corresponding author. Source data were provided in this study.

Reproduced under the paper's license (CC BY), from the paper cited above.

Versions

The history of this record: each version stored by the harvester or made by a correction of its authors or of the maintainers of its code, and what changed in its facts. The texts of the paper (its abstract, its availability statements) are not part of it; versions that changed only those are not listed.

Version 1, 27 September 2026: the first record

Recorded: type, language, journal, volume, issue, pages, dates, 26 authors, 5 keywords, 11 MeSH terms, 3 funders, 74 references.

Cite

This paper

Erel‐Akbaba, G., Park, J., Duhoon, A., Nguyen, C. T. G., Bhaskar, N., Karan, S. K., Akbaba, H., Singh, P., Tran, T. B. T., Zhu, Y., Li, Z., Muhammad, I., Zhang, X., Truong, H. Q., Davidar, A. D., Schmitzer, E., Vattipally, V. N., Lopez, A. F., Kramer, P., . . . Nguyen, T. D. (2026). Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS) in Glioblastoma. Advanced science (Weinheim, Baden-Wurttemberg, Germany), 13(53), e76336. https://doi.org/10.1002/advs.76336

BibTeX

@article{erelakbaba2026biodegradable,
author = {Erel‐Akbaba, Gulsah and Park, Jinyoung and Duhoon, Achal and Nguyen, Cao Thuy Giang and Bhaskar, Nitu and Karan, Sumanta Kumar and Akbaba, Hasan and Singh, Parbeen and Tran, Thi Bao Tram and Zhu, Yuhui and Li, Zhiming and Muhammad, I'jaaz and Zhang, Xiaojun and Truong, Hoang Quan and Davidar, A. Daniel and Schmitzer, Elizabeth and Vattipally, Vikas N. and Lopez, Angelica F. and Kramer, Patrick and Quiroz, Victor M. and Bykov, Ben and Hao, Claire and Manbachi, Amir and Theodore, Nicholas and Doloff, Joshua C. and Nguyen, Thanh D.},
title = {{Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS) in Glioblastoma}},
journal = {Advanced science (Weinheim, Baden-Wurttemberg, Germany)},
year = {2026},
month = jun,
volume = {13},
number = {53},
pages = {e76336},
publisher = {Wiley},
issn = {2198-3844},
doi = {10.1002/advs.76336},
url = {https://doi.org/10.1002/advs.76336},
pmid = {42371791},
pmcid = {PMC13336871}
}

RIS

TY - JOUR
AU - Erel‐Akbaba, Gulsah
AU - Park, Jinyoung
AU - Duhoon, Achal
AU - Nguyen, Cao Thuy Giang
AU - Bhaskar, Nitu
AU - Karan, Sumanta Kumar
AU - Akbaba, Hasan
AU - Singh, Parbeen
AU - Tran, Thi Bao Tram
AU - Zhu, Yuhui
AU - Li, Zhiming
AU - Muhammad, I'jaaz
AU - Zhang, Xiaojun
AU - Truong, Hoang Quan
AU - Davidar, A. Daniel
AU - Schmitzer, Elizabeth
AU - Vattipally, Vikas N.
AU - Lopez, Angelica F.
AU - Kramer, Patrick
AU - Quiroz, Victor M.
AU - Bykov, Ben
AU - Hao, Claire
AU - Manbachi, Amir
AU - Theodore, Nicholas
AU - Doloff, Joshua C.
AU - Nguyen, Thanh D.
TI - Biodegradable Acoustic Targeting for Ultrasound-Supported Gene Therapy (BATUS) in Glioblastoma
T2 - Advanced science (Weinheim, Baden-Wurttemberg, Germany)
J2 - Adv Sci (Weinh)
PY - 2026
DA - 2026/06/29
VL - 13
IS - 53
SP - e76336
SN - 2198-3844
PB - Wiley
DO - 10.1002/advs.76336
UR - https://doi.org/10.1002/advs.76336
LA - en
ER -

CSL-JSON

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