Evidence map›Paper›PMID 29103910›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018

Nanotherapeutics for Gene Modulation that Prevents Apoptosis in the Brain and Fatal Neuroinflammation.

Sang-Soo Kim, Antonina Rait, Emilio R Garrido-Sanabria, Kathleen F Pirollo, Joe B Harford, Esther H Chang

Open access · bronzeAbstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 2 pooled it
0.6field-weighted citation impact, top 33% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

16 citing papers in PubMed, 2 syntheses or guidelines pooled it, 28 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Review
  4. Lipid and polymeric nanocarriers for siRNA delivery to the brain.Journal of controlled release : official journal of the Controlled Release Society · 2025
    Review
  5. Review
  6. Review
  7. Article
  8. Review
  9. Article
  10. Nucleic acid drug vectors for diagnosis and treatment of brain diseases.Signal transduction and targeted therapy · 2023
    Review
  11. Review
  12. Paving the way for small-molecule drug discovery.American journal of translational research · 2021
    Review
  13. Article
  14. Article
  15. Review
  16. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 4 institutions in 1 country.

Sang-Soo KimDepartment of Oncology, Lombardi Comprehensive Cancer Center, Georgetown University, Washington, DC 20057, USA; SynerGene Therapeutics, Inc., Potomac, MD 20854, USA.
Antonina RaitDepartment of Oncology, Lombardi Comprehensive Cancer Center, Georgetown University, Washington, DC 20057, USA.
Emilio R Garrido-SanabriaDepartment of Neurology, Stony Brook University, Stony Brook, NY 11794, USA.
Kathleen F PirolloDepartment of Oncology, Lombardi Comprehensive Cancer Center, Georgetown University, Washington, DC 20057, USA.
Joe B HarfordSynerGene Therapeutics, Inc., Potomac, MD 20854, USA.
Esther H ChangDepartment of Oncology, Lombardi Comprehensive Cancer Center, Georgetown University, Washington, DC 20057, USA. Electronic address: change@georgetown.edu.
Georgetown University Medical Center · USAllogene Therapeutics (United States) · USGeorgetown University · USStony Brook University · US

Funding

Tissue Culture Shared ResourceP30CA051008 · NCI · GEORGETOWN UNIVERSITY · PI STEPHEN W BYERS · 1990 to 2026
$71.5M
Nanoimmunoliposome-Complexed SPIO: Tumor-Specific Detection of Early Lung CancerR01CA132012 · NCI · GEORGETOWN UNIVERSITY · PI CHANG, ESTHER H, FREEDMAN, MATTHEW T · 2008 to 2010
$1.9M
EXTRAMURAL RESEARCH FACILITIES CONSTRUCTION PROJECTSC06RR014567 · NCRR · GEORGETOWN UNIVERSITY · PI PESTELL, RICHARD G · 1999 to 1999
–
NCI NIH HHS P30 CA051008NCI NIH HHS R01 CA132012NCRR NIH HHS C06 RR014567
6 · The paper itself

Abstract

The failure of therapeutic agents to cross the blood-brain barrier (BBB) has been a major impediment in the treatment of neurological disorders and brain tumors. We have addressed this issue using an immunoliposome nanocomplex (designated scL) that delivers therapeutic nucleic acids across the BBB into the deep brain via transcytosis mediated by transferrin receptors. We validated brain delivery of payloads after systemic administration by monitoring uptake of fluorescently labeled payloads and by confirming up- or down-modulation of specific target gene expression in the brain, mainly in neuronal cells. As proof of concept for the therapeutic potential of our delivery system, we employed scL delivering an siRNA targeting tumor necrosis factor alpha to suppress neuroinflammation and neuronal apoptosis and to protect mice in lethal endotoxemia triggered by bacterial lipopolysaccharide. Brain delivery of therapeutic payloads via scL has major implications for the development of treatments for neurological disorders and brain tumors.

Indexed as

Gene Transfer TechniquesTheranostic NanomedicineAnimalsApoptosisBlood-Brain BarrierBrainDisease Models, AnimalDrug Delivery SystemsEncephalitisFemaleGenes, ReporterHumansMiceMice, TransgenicRNA, Small InterferingSuperoxide Dismutase-1RNA, Small InterferingSod1 protein, mouseSuperoxide Dismutase-1Tumor Necrosis Factor-alphaCNS drug deliverycrossing blood-brain barrierneuroinflammationreceptor-mediated transcytosissiRNA deliverytargeted nanocomplextherapeutic nucleic acids

Identifiers

PMID29103910
PMCPMC5763150
OpenAlexW2761220582

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.