Evidence map›Paper›PMID 37587230›Full record

ArticleGene therapy2024

Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates.

Tricia H Burdo, Chen Chen, Rafal Kaminski, Ilker K Sariyer, Pietro Mancuso, Martina Donadoni, Mandy D Smith, Rahsan Sariyer, Maurizio Caocci, Shuren Liao and 11 more

Erratum issuedOpen access · hybridAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in 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 28 papers.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed
5.1field-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

28 citing papers in PubMed, 33 citations in OpenAlex.

  1. Review
  2. Molecular therapy. Nucleic acids · 2026
    Article
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  12. Article
  13. CRISPR/Cas9 for achieving postintervention HIV control.Current opinion in HIV and AIDS · 2025
    Review
  14. FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
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  16. Article
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

21 authors at 3 institutions in 1 country.

Tricia H BurdoDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA. burdot@temple.edu.
Chen ChenDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Rafal KaminskiDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.ORCID 0000-0001-8827-6658
Ilker K SariyerDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Pietro MancusoDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.ORCID 0000-0001-7079-9848
Martina DonadoniDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Mandy D SmithDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Rahsan SariyerDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Maurizio CaocciDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Shuren LiaoDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Hong LiuDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
Wenwen HuoExcision BioTherapeutics, Inc., San Francisco, CA, USA.
Huaqing ZhaoCenter for Biostatistics and Epidemiology, Department of Biomedical Education and Data Science, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA.
John MisamoreBioQual, Inc., Rockville, MD, USA.
Mark G LewisBioQual, Inc., Rockville, MD, USA.
Vahan SimonyanEmbleema, Metuchen, NJ, USA.
Elaine E ThompsonEmbleema, Metuchen, NJ, USA.
Ethan Y XuExcision BioTherapeutics, Inc., San Francisco, CA, USA.
Thomas J CradickExcision BioTherapeutics, Inc., San Francisco, CA, USA.ORCID 0000-0002-8489-8318
Jennifer GordonExcision BioTherapeutics, Inc., San Francisco, CA, USA. jgordon@excisionbio.com.
Kamel KhaliliDepartment of Microbiology, Immunology, and Inflammation, Center for NeuroVirology and Gene Editing, Lewis Katz School of Medicine, Temple University, Philadelphia, PA, 19140, USA. kkhalili@temple.edu.ORCID 0000-0002-6819-5217
Temple University · USBioqual · USAtara Biotherapeutics (United States) · US

Funding

Viral TestingU42OD021458 · OD · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI Armando Gil Burgos-Rodriguez, Marcos Lopez · 2016 to 2026
$45.1M
Translational Science InitiativeP40OD012217 · OD · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI Melween I. Martinez · 2012 to 2026
$40.4M
Viral Gene Editing and Bioinformatics Core for Institution # 269291P30MH092177 · NIMH · TEMPLE UNIV OF THE COMMONWEALTH · PI Ilker Kudret Sariyer · 2011 to 2026
$24.9M
NIH HHS P40 OD012217NIH HHS U42 OD021458NIMH NIH HHS P30 MH092177
6 · The paper itself

Abstract

In this study, we demonstrate the safety and utility of CRISPR-Cas9 gene editing technology for in vivo editing of proviral DNA in ART-treated, virally controlled simian immunodeficiency virus (SIV) infected rhesus macaques, an established model for HIV infection. EBT-001 is an AAV9-based vector delivering SaCas9 and dual guide RNAs designed to target multiple regions of the SIV genome: the viral LTRs, and the Gag gene. The results presented here demonstrate that a single IV inoculation of EBT-001 at each of 3 dose levels (1.4 × 10

Indexed as

CRISPR-Cas SystemsGene EditingMacaca mulattaSimian Acquired Immunodeficiency SyndromeSimian Immunodeficiency VirusAnimalsDependovirusGenetic TherapyGenetic VectorsTissue Distribution

Identifiers

PMID37587230
PMCPMC11090835
OpenAlexW4385852584

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.