Evidence map›Paper›PMID 32161326›Full record

ArticleScientific reports2020

In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery.

Cort B Breuer, Killian S Hanlon, Jeya-Shree Natasan, Adrienn Volak, Amine Meliani, Federico Mingozzi, Benjamin P Kleinstiver, James J Moon, Casey A Maguire

Open access · goldAbstract read
In one paragraph

Article in Scientific reports, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed
3.6field-weighted citation impact, top 7% 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

21 citing papers in PubMed, 29 citations in OpenAlex.

  1. Article
  2. Article
  3. The curious case of AAV immunology.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  4. Article
  5. Targeted gene delivery systems for T-cell engineering.Cellular oncology (Dordrecht, Netherlands) · 2024
    Review
  6. In vivo gene delivery to immune cells.Current opinion in biotechnology · 2024
    Review
  7. Article
  8. Article
  9. Article
  10. Review
  11. Article
  12. Article
  13. Review
  14. Article
  15. Article
  16. Vector Strategies to Actualize B Cell-Based Gene Therapies.Journal of immunology (Baltimore, Md. : 1950) · 2021
    Review
  17. Review
  18. Review
  19. CRISPR/Cas-Dependent and Nuclease-FreeHuman gene therapy · 2021
    Review
  20. Article
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

9 authors at 3 institutions in 2 countries.

Cort B BreuerCenter for Immunology and Inflammatory Diseases, Massachusetts General Hospital, Charlestown, MA, USA.
Killian S HanlonDepartment of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.
Jeya-Shree NatasanDepartment of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.
Adrienn VolakDepartment of Neurology, Massachusetts General Hospital, Charlestown, MA, USA.
Amine MelianiGenethon, INSERM UMR S951, Evry, France.
Federico MingozziGenethon, INSERM UMR S951, Evry, France.
Benjamin P KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital, Boston, MA, USA.ORCID http://orcid.org/0000-0002-5469-0655
James J Moon *Center for Immunology and Inflammatory Diseases, Massachusetts General Hospital, Charlestown, MA, USA. jjmoon@mgh.harvard.edu.ORCID http://orcid.org/0000-0001-8246-931X
Casey A Maguire *Department of Neurology, Massachusetts General Hospital, Charlestown, MA, USA. cmaguire@mgh.harvard.edu.
Harvard University · USMassachusetts General Hospital · USInserm · FR

Funding

PROJECT 4: Somatic evolution of the hematopoietic system in cardiovascular diseaseP01HL142494 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Matthias Nahrendorf · 2019 to 2026
$19.5M
Optimizing Cancer Immunotherapy Safety and Efficacy using Genome EditingR00CA218870 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI KLEINSTIVER, BENJAMIN PETER · 2018 to 2020
$730k
NCI NIH HHS R00 CA218870NHLBI NIH HHS P01 HL142494
6 · The paper itself

Abstract

Ex-vivo gene therapy using stem cells or T cells transduced by retroviral or lentiviral vectors has shown remarkable efficacy in the treatment of immunodeficiencies and cancer. However, the process is expensive, technically challenging, and not readily scalable to large patient populations, particularly in underdeveloped parts of the world. Direct in vivo gene therapy would avoid these issues, and such approaches with adeno-associated virus (AAV) vectors have been shown to be safe and efficacious in clinical trials for diseases affecting differentiated tissues such as the liver and CNS. However, the ability to transduce lymphocytes with AAV in vivo after systemic delivery has not been carefully explored. Here, we show that both standard and exosome-associated preparations of AAV8 vectors can effectively transduce a variety of immune cell populations including CD4

Indexed as

Genetic EngineeringGene Transfer TechniquesAnimalsDependovirusExosomesGenetic VectorsGreen Fluorescent ProteinsMaleMiceMice, Inbred C57BLT-LymphocytesTransduction, GeneticTransgenesGreen Fluorescent Proteins

Identifiers

PMID32161326
PMCPMC7066196
OpenAlexW3012467628

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.