Evidence map›Paper›PMID 35780495›Full record

ArticleAdvanced science (Weinheim, Baden-Wurttemberg, Germany)2022

Red Blood Cell Anchoring Enables Targeted Transduction and Re-Administration of AAV-Mediated Gene Therapy.

Zongmin Zhao, Jayoung Kim, Vinny Chandran Suja, Neha Kapate, Yongsheng Gao, Junling Guo, Vladimir R Muzykantov, Samir Mitragotri

Open access · goldAbstract read
In one paragraph

Article in Advanced science (Weinheim, Baden-Wurttemberg, Germany), 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
5.4field-weighted citation impact, top 4% 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

15 citing papers in PubMed, 32 citations in OpenAlex.

  1. Article
  2. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  3. Review
  4. Article
  5. Review
  6. Review
  7. Article
  8. Delivering living medicines with biomaterials.Nature reviews. Materials · 2025
    Article
  9. Review
  10. Article
  11. Review
  12. Review
  13. Red blood cells: a potential delivery system.Journal of nanobiotechnology · 2023
    Review
  14. Article
  15. 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

8 authors at 2 institutions in 1 country.

Zongmin ZhaoJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.
Jayoung KimJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.
Vinny Chandran SujaJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.
Neha KapateJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.
Yongsheng GaoJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.
Junling GuoJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.
Vladimir R MuzykantovDepartment of Systems Pharmacology and Translational Therapeutics and Center for Translational Targeted Therapeutics and Nanomedicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, USA.
Samir MitragotriJohn A. Paulson School of Engineering and Applied Sciences, Harvard University, Cambridge, MA, 02138, USA.ORCID 0000-0002-2459-8305
Harvard University · USTarget (United States) · US

Funding

School of Engineering and Applied SciencesWyss Institute at Harvard University
6 · The paper itself

Abstract

Adeno-associated virus (AAV)-mediated gene therapy is a promising therapeutic modality for curing many diseases including monogenic diseases. However, limited tissue-targeting and restricted re-administration due to the vector immunogenicity largely restrict its therapeutic potential. Here, using a red blood cell (RBC) as the carrier vehicle for AAV is demonstrated to improve its tissue-targeted transduction and enable its re-administration. Anchoring AAV to the RBC surface minimally affected its infectability toward endothelial cells. Meanwhile, AAV anchored onto RBCs is predominantly delivered to and shows efficient transduction in the lungs by virtue of the biophysical features of RBCs. RBC-anchored AAVs lead to a four- to five-fold enhancement in target gene expression in the lungsas compared to free AAVs following a single- or dual-dosing regimen. While RBC anchoring does not prevent the induction of adaptive immune responses against AAV, it results in successful transgene expression upon re-administration following prior AAV exposure. The ability to re-administer is partially attributed to the delayed and reduced AAV neutralization by neutralizing antibodies, resulting from the combination of limited exposure of physically confined AAVs and the short time required to reach the lungs. This study's findings suggest that the RBC-mediated approach is a promising strategy for repetitive, targeted AAV gene therapy.

Indexed as

DependovirusGenetic VectorsEndothelial CellsErythrocytesGenetic Therapyadeno-associated virusgene therapyimmunomodulationneutralizing antibodyred blood cell hitchhiking

Identifiers

PMID35780495
PMCPMC9404386
OpenAlexW4283785000

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.