Evidence map›Paper›PMID 39091030›Full record

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

A platform to deliver single and bi-specific Cas9/guide RNA to perturb genes in vitro and in vivo.

Yi-Jia Li, Sheng-Hsuan Chien, Rui Huang, Andreas Herrmann, Qianqian Zhao, Pei-Chuan Li, Chunyan Zhang, Antons Martincuks, Nicole Lugo Santiago, Katherine Zong and 7 more

Abstract read
PubMed Full text at PMC Publisher
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. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed
–field-weighted citation impact
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

5 citing papers in PubMed.

  1. Review
  2. Article
  3. Article
  4. Review
  5. 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

17 authors.

Yi-Jia LiDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA. Electronic address: yili@coh.org.
Sheng-Hsuan ChienCellular Immunotherapy Center, Department of Hematology and Hematopoietic Cell Transplantation, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA; Division of Transfusion Medicine, Department of Medicine, Taipei Veterans General Hospital, and Institute of Clinical Medicine, National Yang-Ming Chiao Tung University, Taipei 11201, Taiwan.
Rui HuangDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA.
Andreas HerrmannDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA.
Qianqian ZhaoDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA.
Pei-Chuan LiDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA.
Chunyan ZhangCellular Immunotherapy Center, Department of Hematology and Hematopoietic Cell Transplantation, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA.
Antons MartincuksDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA.
Nicole Lugo SantiagoDepartment of Surgery, Division of Gynecologic Oncology, City of Hope National Medical Center, Duarte, CA 91010, USA.
Katherine ZongDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA.
Piotr SwiderskiDNA/RNA Synthesis Laboratory, Beckman Research Institute at City of Hope Comprehensive Cancer Center, Duarte, CA 91010, USA.
Ross A OkimotoDepartment of Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Helen Diller Family Comprehensive Cancer Center, University of California, San Francisco, San Francisco, CA 94115, USA.
Mihae SongDepartment of Surgery, Division of Gynecologic Oncology, City of Hope National Medical Center, Duarte, CA 91010, USA.
Lorna RodriguezDepartment of Surgery, Division of Gynecologic Oncology, City of Hope National Medical Center, Duarte, CA 91010, USA.
Stephen J FormanCellular Immunotherapy Center, Department of Hematology and Hematopoietic Cell Transplantation, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA.
Xiuli WangCellular Immunotherapy Center, Department of Hematology and Hematopoietic Cell Transplantation, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA.
Hua YuDepartment of Immuno-Oncology, Beckman Research Institute and City of Hope Medical Center, Duarte, CA 91010, USA. Electronic address: hyu@coh.org.

Funding

Transplant for Lymphoma:Therapy-Related LeukemiaP50CA107399 · NCI · CITY OF HOPE/BECKMAN RESEARCH INSTITUTE · PI FORMAN, STEPHEN J, KWAK, LARRY W · 2004 to 2022
$35.5M
NCI NIH HHS P50 CA107399
6 · The paper itself

Abstract

Although CRISPR-Cas9 technology is poised to revolutionize the treatment of diseases with underlying genetic mutations, it faces some significant issues limiting clinical entry. They include low-efficiency in vivo systemic delivery and undesired off-target effects. Here, we demonstrate, by modifying Cas9 with phosphorothioate-DNA oligos (PSs), that one can efficiently deliver single and bi-specific CRISPR-Cas9/guide RNA (gRNA) dimers in vitro and in vivo with reduced off-target effects. We show that PS-Cas9/gRNA-mediated gene knockout preserves chimeric antigen receptor T cell viability and expansion in vitro and in vivo. PS-Cas9/gRNA mediates gene perturbation in patient-derived tumor organoids and mouse xenograft tumors, leading to potent tumor antitumor effects. Further, HER2 antibody-PS-Cas9/gRNA conjugate selectively perturbs targeted genes in HER2

Indexed as

CRISPR-Cas SystemsRNA, Guide, CRISPR-Cas SystemsXenograft Model Antitumor AssaysAnimalsCell Line, TumorCRISPR-Associated Protein 9Erb-b2 Receptor Tyrosine KinasesFemaleGene EditingGene Knockout TechniquesHumansMiceOvarian NeoplasmsCRISPR-Associated Protein 9Erb-b2 Receptor Tyrosine KinasesRNA, Guide, CRISPR-Cas Systemsbi-specificCAR-TCas9deliveryoff-target effectsovarian cancer

Identifiers

PMID39091030
PMCPMC11489542

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.