Evidence map›Paper›PMID 28956530›Full record

ArticleeLife2017

Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.

Rasmus O Bak, Daniel P Dever, Andreas Reinisch, David Cruz Hernandez, Ravindra Majeti, Matthew H Porteus

Erratum issuedOpen access · goldAbstract read
In one paragraph

Article in eLife, 2017. 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 78 papers.

0numbers the graph read from it
0cells of the map it votes in
78citing papers in PubMed
6.0field-weighted citation impact, top 3% 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

78 citing papers in PubMed, 125 citations in OpenAlex.

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  12. Gene therapy then and now: A look back at changes in the field over the past 25 years.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
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18 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 1 institution in 1 country.

Rasmus O Bak *Department of Pediatrics, Stanford University, Stanford, United States.ORCID https://orcid.org/0000-0002-7383-0297
Daniel P Dever *Department of Pediatrics, Stanford University, Stanford, United States.
Andreas Reinisch *Department of Medicine, Division of Hematology, Stanford University, Stanford, United States.ORCID http://orcid.org/0000-0001-9333-7689
David Cruz HernandezDepartment of Medicine, Division of Hematology, Stanford University, Stanford, United States.
Ravindra MajetiDepartment of Medicine, Division of Hematology, Stanford University, Stanford, United States.
Matthew H PorteusDepartment of Pediatrics, Stanford University, Stanford, United States.ORCID http://orcid.org/0000-0002-3850-4648
Stanford University · US

Funding

Nanomedicine Center for Nucleoprotein MachinesPN2EY018244 · NEI · RICE UNIVERSITY · PI BAO, GANG · 2006 to 2015
$24.4M
Genome Editing by Homologous Recombination to Create HIV Resistant Immune SystemR01AI120766 · NIAID · STANFORD UNIVERSITY · PI PORTEUS, MATTHEW H · 2015 to 2019
$2.0M
Pre-Clinical Development of Nuclease Mediated Gene Therapy for SCIDR01AI097320 · NIAID · STANFORD UNIVERSITY · PI PORTEUS, MATTHEW H · 2013 to 2017
$1.9M
Pre-Leukemic Hematopoietic Stem Cells and Clonal Evolution in Human AMLR01CA188055 · NCI · STANFORD UNIVERSITY · PI MAJETI, RAVINDRA · 2014 to 2018
$1.6M
Austrian Science Fund FWF J 3358NCI NIH HHS R01 CA188055NEI NIH HHS PN2 EY018244NIAID NIH HHS R01 AI097320NIAID NIH HHS R01 AI120766
6 · The paper itself

Abstract

Precise and efficient manipulation of genes is crucial for understanding the molecular mechanisms that govern human hematopoiesis and for developing novel therapies for diseases of the blood and immune system. Current methods do not enable precise engineering of complex genotypes that can be easily tracked in a mixed population of cells. We describe a method to multiplex homologous recombination (HR) in human hematopoietic stem and progenitor cells and primary human T cells by combining rAAV6 donor delivery and the CRISPR/Cas9 system delivered as ribonucleoproteins (RNPs). In addition, the use of reporter genes allows FACS-purification and tracking of cells that have had multiple alleles or loci modified by HR. We believe this method will enable broad applications not only to the study of human hematopoietic gene function and networks, but also to perform sophisticated synthetic biology to develop innovative engineered stem cell-based therapeutics.

Indexed as

CRISPR-Cas SystemsHomologous RecombinationCells, CulturedFlow CytometryGene EditingGenes, ReporterHematopoietic Stem CellsHumansT-LymphocytesCRISPR/Cas9developmental biologyevolutionary biologygene editinggenomicshematopoietic stem cellshumanmousemultiplexstem cells

Identifiers

PMID28956530
PMCPMC5656432
OpenAlexW2759699238

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.