Evidence map›Paper›PMID 38352420›Full record

ArticlebioRxiv : the preprint server for biology2024

Single-Stranded DNA with Internal Base Modifications Mediates Highly Efficient Gene Insertion in Primary Cells.

Karen L Kanke, Rachael E Rayner, Eli Abel, Aparna Venugopalan, Ma Suu, Jacob T Stack, Reza Nouri, Gongbo Guo, Tatyana A Vetter, Estelle Cormet-Boyaka and 2 more

Open access · greenAbstract readPreprint
PubMed Full text at PMC Publisher
In one paragraph

Article in bioRxiv : the preprint server for biology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed, 1 citations in OpenAlex.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 2 institutions in 1 country.

Karen L KankeCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Rachael E RaynerDepartment of Veterinary Biosciences, The Ohio State University, Columbus, OH.
Eli AbelCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Aparna VenugopalanCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Ma SuuCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Jacob T StackCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Reza NouriCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Gongbo GuoInstitute for Genomic Medicine, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Tatyana A VetterCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Estelle Cormet-BoyakaDepartment of Veterinary Biosciences, The Ohio State University, Columbus, OH.
Mark E HesterInstitute for Genomic Medicine, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Sriram VaidyanathanCenter for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH.
Nationwide Children's Hospital · USThe Ohio State University · US

Funding

The OSU Center for Clinical and Translational Science: Advancing Today's Discoveries to Improve HealthUL1TR002733 · NCATS · OHIO STATE UNIVERSITY · PI RINGEL, MATTHEW D · 2018 to 2022
$28.9M
Stem Cell and Transplantation BiologyU54DK106829 · NIDDK · FRED HUTCHINSON CANCER RESEARCH CENTER · PI DEREK L STIREWALT · 2015 to 2026
$9.1M
A universal genome editing strategy to develop an airway stem cell therapy for cystic fibrosisR00HL151900 · NHLBI · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI VAIDYANATHAN, SRIRAM · 2022 to 2024
$747k
A universal genome editing strategy to develop an airway stem cell therapy for cystic fibrosisK99HL151900 · NHLBI · STANFORD UNIVERSITY · PI VAIDYANATHAN, SRIRAM · 2021 to 2022
$333k
NCATS NIH HHS UL1 TR002733NHLBI NIH HHS K99 HL151900NHLBI NIH HHS R00 HL151900NIDDK NIH HHS U54 DK106829
6 · The paper itself

Abstract

Single-stranded DNA (ssDNA) templates along with Cas9 have been used for gene insertion but suffer from low efficiency. Here, we show that ssDNA with chemical modifications in 10-17% of internal bases (eDNA) is compatible with the homologous recombination machinery. Moreover, eDNA templates improve gene insertion by 2-3 fold compared to unmodified and end-modified ssDNA in airway basal stem cells (ABCs), hematopoietic stem and progenitor cells (HSPCs), T-cells and endothelial cells. Over 50% of alleles showed gene insertion in three clinically relevant loci (

Identifiers

PMID38352420
PMCPMC10862822
OpenAlexW4391481276

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.