Evidence map›Paper›PMID 28267358›Full record

ArticleHuman gene therapy methods2017

RNA Trans-Splicing Targeting Endogenous β-Globin Pre-Messenger RNA in Human Erythroid Cells.

Naoya Uchida, Kareem N Washington, Brian Mozer, Charlotte Platner, Josiah Ballantine, Luke P Skala, Lydia Raines, Anna Shvygin, Matthew M Hsieh, Lloyd G Mitchell and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy methods, 2017. 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. Article
  2. RNA exon editing: Splicing the way to treat human diseases.Molecular therapy. Nucleic acids · 2024
    Review
  3. Therapeutic applications of trans-splicing.British medical bulletin · 2020
    Article
  4. Article
  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

11 authors.

Naoya Uchida1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Kareem N Washington2 Genetics and Human Genetics, Howard University , Washington, DC.
Brian Mozer3 Office of Research Integrity , Office of the Assistant Secretary for Health, Rockville, Maryland.
Charlotte Platner1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Josiah Ballantine1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Luke P Skala1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Lydia Raines1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Anna Shvygin1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Matthew M Hsieh1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.
Lloyd G Mitchell4 RetroTherapy , Bethesda, Maryland.
John F Tisdale1 Molecular and Clinical Hematology Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) , Bethesda, Maryland.

Funding

Development of tools to modify globin gene expression in stem cellsR43HL106982 · NHLBI · RETROTHERAPY, LLC · PI MITCHELL, LLOYD G · 2011 to 2011
$521k
NHLBI NIH HHS R43 HL106982
6 · The paper itself

Abstract

Sickle cell disease results from a point mutation in exon 1 of the β-globin gene (total 3 exons). Replacing sickle β-globin exon 1 (and exon 2) with a normal sequence by trans-splicing is a potential therapeutic strategy. Therefore, this study sought to develop trans-splicing targeting β-globin pre-messenger RNA among human erythroid cells. Binding domains from random β-globin sequences were comprehensively screened. Six candidates had optimal binding, and all targeted intron 2. Next, lentiviral vectors encoding RNA trans-splicing molecules were constructed incorporating a unique binding domain from these candidates, artificial 5' splice site, and γ-globin cDNA, and trans-splicing was evaluated in CD34

Indexed as

Genetic TherapyAnemia, Sickle Cellbeta-GlobinsErythroid CellsExonsGenetic VectorsHumansIntronsPoint MutationRNA PrecursorsRNA SplicingTransfectionTrans-Splicingbeta-GlobinsRNA Precursorslentiviral vectorRNA trans-splicingβ-globin gene

Identifiers

PMID28267358
PMCPMC5397226

What OpenQuestion holds

Textmetadata
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Registered trials

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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.