Evidence map›Paper›PMID 36210364›Full record

ArticleNature communications2022

Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing.

Trevor J Gonzalez, Katherine E Simon, Leo O Blondel, Marco M Fanous, Angela L Roger, Maribel Santiago Maysonet, Garth W Devlin, Timothy J Smith, Daniel K Oh, L Patrick Havlik and 5 more

Open access · goldAbstract read
In one paragraph

Article in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.

0numbers the graph read from it
0cells of the map it votes in
62citing papers in PubMed
5.9field-weighted citation impact, top 2% 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

62 citing papers in PubMed, 76 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Review
  5. Article
  6. Review
  7. AAVcc47-The Yale journal of biology and medicine · 2026
    Article
  8. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  9. Article
  10. Enhancer-directed gene delivery for digit regeneration based on conserved epidermal factors.Proceedings of the National Academy of Sciences of the United States of America · 2026
    Article
  11. Article
  12. Article
  13. Epigenetic editing: from concept to clinic.Nature reviews. Drug discovery · 2026
    Review
  14. Mechanisms and Therapeutic Potential of Human Cardiomyocyte Proliferation.Journal of cardiovascular development and disease · 2026
    Review
  15. Article
  16. Article
  17. Review
  18. Article
  19. Article
  20. Article

2 more citing papers are in PubMed but not listed here.

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

15 authors at 2 institutions in 1 country.

Trevor J GonzalezDepartment of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC, USA.ORCID http://orcid.org/0000-0002-2242-4483
Katherine E Simon *Department of Surgery, Duke University School of Medicine, Durham, NC, USA.
Leo O Blondel *Department of Surgery, Duke University School of Medicine, Durham, NC, USA.ORCID http://orcid.org/0000-0002-9211-259X
Marco M FanousDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
Angela L RogerDepartment of Pediatrics, Duke University School of Medicine, Durham, NC, USA.ORCID http://orcid.org/0000-0001-5249-7176
Maribel Santiago MaysonetStrideBio Inc., Research Triangle Park, Durham, NC, USA.
Garth W DevlinDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
Timothy J SmithDepartment of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC, USA.
Daniel K OhDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
L Patrick HavlikDepartment of Surgery, Duke University School of Medicine, Durham, NC, USA.
Ruth M Castellanos RiveraStrideBio Inc., Research Triangle Park, Durham, NC, USA.
Jorge A PiedrahitaNorth Carolina State University College of Veterinary Medicine, Raleigh, NC, USA.
Mai K ElMallahDepartment of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Charles A GersbachDuke Regeneration Center, Duke University School of Medicine, Durham, NC, USA.ORCID http://orcid.org/0000-0003-1478-4013
Aravind AsokanDepartment of Molecular Genetics and Microbiology, Duke University School of Medicine, Durham, NC, USA. aravind.asokan@duke.edu.ORCID http://orcid.org/0000-0001-5563-4877
Duke University · USNorth Carolina State University · US

Funding

University Training Program in Biomolecular and Tissue EngineeringT32GM144291 · NIGMS · DUKE UNIVERSITY · PI Charles A. Gersbach, Tatiana Segura · 2022 to 2026
$2.6M
Evolving High Potency AAV Vectors for Neuromuscular Genome EditingUH3AR075336 · NIAMS · DUKE UNIVERSITY · PI ASOKAN, ARAVIND · 2021 to 2022
$2.0M
Targeted Therapy for Pompe DiseaseR01HD099486 · NICHD · DUKE UNIVERSITY · PI ELMALLAH, MAI · 2019 to 2023
$1.7M
Epigenome Editing Technologies for Treating Diverse DiseaseU01AI146356 · NIAID · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2019 to 2022
$1.6M
NIAID NIH HHS U01 AI146356NIAMS NIH HHS UH3 AR075336NICHD NIH HHS R01 HD099486NIGMS NIH HHS T32 GM144291
6 · The paper itself

Abstract

Recombinant adeno-associated viral (AAV) vectors are a promising gene delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window. Effective clinical translation is confounded, at least in part, by differences in AAV biology across animal species. Here, we tackle this challenge by sequentially evolving AAV capsid libraries in mice, pigs and macaques. We discover a highly potent, cross-species compatible variant (AAV.cc47) that shows improved attributes benchmarked against AAV serotype 9 as evidenced by robust reporter and therapeutic gene expression, Cre recombination and CRISPR genome editing in normal and diseased mouse models. Enhanced transduction efficiency of AAV.cc47 vectors is further corroborated in macaques and pigs, providing a strong rationale for potential clinical translation into human gene therapies. We envision that ccAAV vectors may not only improve predictive modeling in preclinical studies, but also clinical translatability by broadening the therapeutic window of AAV based gene therapies.

Indexed as

DependovirusGene EditingAnimalsGenetic TherapyGenetic VectorsHumansMacacaMiceSwineTransduction, Genetic

Identifiers

PMID36210364
PMCPMC9548504
OpenAlexW4303982751

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.