Evidence map›Paper›PMID 37919899›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2024

Targeted, safe, and efficient gene delivery to human hematopoietic stem and progenitor cells in vivo using the engineered AVID adenovirus vector platform.

Jia Yao, Svetlana Atasheva, Nicole Wagner, Nelson C Di Paolo, Phoebe L Stewart, Dmitry M Shayakhmetov

Erratum issuedOpen access · hybridAbstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
5.3field-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

10 citing papers in PubMed, 17 citations in OpenAlex.

  1. Review
  2. Molecular therapy. Methods & clinical development · 2025
    Article
  3. Review
  4. Molecular therapy. Methods & clinical development · 2024
    Article
  5. Molecular Engineering of Virus Tropism.International journal of molecular sciences · 2024
    Review
  6. Auto-expansion ofMolecular therapy. Methods & clinical development · 2024
    Article
  7. Article
  8. Review
  9. Viral platform engineering for targeted gene delivery to human hematopoietic stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  10. Review
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 2 institutions in 1 country.

Jia YaoLowance Center for Human Immunology, Departments of Pediatrics and Medicine, Emory University School of Medicine, Atlanta, GA 30322, USA.
Svetlana AtashevaLowance Center for Human Immunology, Departments of Pediatrics and Medicine, Emory University School of Medicine, Atlanta, GA 30322, USA.
Nicole WagnerCleveland Center for Membrane and Structural Biology, Department of Pharmacology, Case Western Reserve University, Cleveland, OH 44106, USA.
Nelson C Di PaoloAdCure Bio, LLC, Century Spring West, 6000 Lake Forrest Drive, Atlanta, GA 30328, USA.
Phoebe L StewartCleveland Center for Membrane and Structural Biology, Department of Pharmacology, Case Western Reserve University, Cleveland, OH 44106, USA.
Dmitry M ShayakhmetovLowance Center for Human Immunology, Departments of Pediatrics and Medicine, Emory University School of Medicine, Atlanta, GA 30322, USA; Emory Vaccine Center, Emory University School of Medicine, Atlanta, GA 30322, USA; Discovery and Developmental Therapeutics Program, Winship Cancer Institute of Emory University, Atlanta, GA 30322, USA. Electronic address: dmitryshay@emory.edu.
Emory University · USCase Western Reserve University · US

Funding

Winship Cancer Institute Cancer Center Support GrantP30CA138292 · NCI · EMORY UNIVERSITY · PI Ragini Reiney Kudchadkar · 2009 to 2026
$47.5M
Mechanisms of adenovirus neutralizationR01AI107960 · NIAID · EMORY UNIVERSITY · PI SHAYAKHMETOV, DMITRY, STEWART, PHOEBE L · 2014 to 2024
$6.5M
Mechanistic factors limiting utility of adenovirus vectors for treatment of neoplaR01CA267694 · NCI · EMORY UNIVERSITY · PI Dmitry Shayakhmetov · 2022 to 2026
$3.1M
Molecular mechanisms of adenovirus neutralizationR37AI107960 · NIAID · EMORY UNIVERSITY · PI PHOEBE L STEWART, Dmitry Shayakhmetov · 2026 to 2026
$806k
NCI NIH HHS P30 CA138292NCI NIH HHS R01 CA267694NIAID NIH HHS R01 AI107960NIAID NIH HHS R37 AI107960
6 · The paper itself

Abstract

Targeted delivery and cell-type-specific expression of gene-editing proteins in various cell types in vivo represent major challenges for all viral and non-viral delivery platforms developed to date. Here, we describe the development and analysis of artificial vectors for intravascular delivery (AVIDs), an engineered adenovirus-based gene delivery platform that allows for highly targeted, safe, and efficient gene delivery to human hematopoietic stem and progenitor cells (HSPCs) in vivo after intravenous vector administration. Due to a set of refined structural modifications, intravenous administration of AVIDs did not trigger cytokine storm, hepatotoxicity, or thrombocytopenia. Single intravenous administration of AVIDs to humanized mice, grafted with human CD34

Indexed as

Hematopoietic Stem CellsHematopoietic Stem Cell TransplantationAdenoviridaeAnimalsAntigens, CD34Genetic TherapyGene Transfer TechniquesHumansMiceAntigens, CD34adenovirusgene therapyhematopoietic stem cellsintravenous administrationtargeted in vivo deliveryvector engineeringvector targeting

Identifiers

PMID37919899
PMCPMC10787117
OpenAlexW4388153915

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.