Evidence map›Paper›PMID 25230987›Full record

ArticleScientific reports2014

Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases.

Ulrike Mock, Kristoffer Riecken, Belinda Berdien, Waseem Qasim, Emma Chan, Toni Cathomen, Boris Fehse

Open access · goldAbstract read
In one paragraph

Article in Scientific reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.

0numbers the graph read from it
0cells of the map it votes in
41citing papers in PubMed
3.6field-weighted citation impact, top 6% 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

41 citing papers in PubMed, 70 citations in OpenAlex.

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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

7 authors at 4 institutions in 2 countries.

Ulrike MockResearch Dept. Cell and Gene Therapy, Clinic for Stem Cell Transplantation, University Medical Centre Hamburg-Eppendorf, 20246 Hamburg, Germany.
Kristoffer RieckenResearch Dept. Cell and Gene Therapy, Clinic for Stem Cell Transplantation, University Medical Centre Hamburg-Eppendorf, 20246 Hamburg, Germany.
Belinda BerdienResearch Dept. Cell and Gene Therapy, Clinic for Stem Cell Transplantation, University Medical Centre Hamburg-Eppendorf, 20246 Hamburg, Germany.
Waseem QasimMolecular Immunology Unit, UCL Institute of Child Health, London, WC1N 1EH, United Kingdom.
Emma ChanMolecular Immunology Unit, UCL Institute of Child Health, London, WC1N 1EH, United Kingdom.
Toni Cathomen1] Institute for Cell and Gene Therapy, University Medical Centre Freiburg, Hugstetter Str. 55, 79106 Freiburg, Germany [2] Centre for Chronic Immunodeficiency, University Medical Centre Freiburg, Hugstetter Str. 55, 79106 Freiburg, Germany.
Boris FehseResearch Dept. Cell and Gene Therapy, Clinic for Stem Cell Transplantation, University Medical Centre Hamburg-Eppendorf, 20246 Hamburg, Germany.
Universität Hamburg · DEUniversity College London · GBUniversity Medical Center Freiburg · DEUniversity Medical Center Hamburg-Eppendorf · DE

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

TAL-effector nucleases (TALENs) are attractive tools for sequence-specific genome modifications, but their delivery still remains problematic. It is well known that the presence of multiple sequence repeats in TALEN genes hampers the use of lentiviral vectors. We report that lentiviral vectors readily package full-length vector mRNAs encoding TALENs, but recombination during reverse transcription prevents successful delivery. We reasoned that preventing reverse transcription of lentiviral-vector RNA would allow transfer of TALENs as mRNA. We demonstrate that lentiviral particles containing genetically inactivated reverse transcriptase (RT) mediated efficient transduction of cultured cells and supported transient transgene expression. For proof-of-principle, we transferred CCR5- and TCR-specific TALEN pairs for efficient targeted genome editing and abrogated expression for each of the receptor proteins in different cell lines. Combining the high specificity of TALENs with efficient lentiviral gene delivery should advance genome editing in vitro and potentially in vivo, and RT-deficient lentiviral vectors may be useful for transient expression of various other genes-of-interest.

Indexed as

Genetic EngineeringGenetic VectorsDrug Delivery SystemsEndonucleasesGene Expression RegulationGene Transfer TechniquesGenome, HumanHEK293 CellsHIV Reverse TranscriptaseHumansLentivirusMutationReceptors, CCR5RNA, MessengerTransduction, GeneticEndonucleasesHIV Reverse TranscriptaseReceptors, CCR5RNA, Messenger

Identifiers

PMID25230987
PMCPMC4166709
OpenAlexW2073742601

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.