Evidence map›Paper›PMID 34477013›Full record

ArticleHuman gene therapy2021

Investigating the Barrier Activity of Novel, Human Enhancer-Blocking Chromatin Insulators for Hematopoietic Stem Cell Gene Therapy.

Penelope-Georgia Papayanni, Nikoletta Psatha, Panayota Christofi, Xing-Guo Li, Pamela Melo, Monica Volpin, Eugenio Montini, Mingdong Liu, Georgios Kaltsounis, Minas Yiangou and 6 more

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

Article in Human gene therapy, 2021. 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
0.7field-weighted citation impact, top 26% 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

5 citing papers in PubMed, 7 citations in OpenAlex.

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

16 authors at 7 institutions in 4 countries.

Penelope-Georgia PapayanniHematopoietic Cell Transplantation Unit, Hematology Department, Gene and Cell Therapy Center, "George Papanikolaou" Hospital, Thessaloniki, Greece.
Nikoletta PsathaAltius Institute for Biomedical Sciences, Seattle, Washington, USA.
Panayota ChristofiHematopoietic Cell Transplantation Unit, Hematology Department, Gene and Cell Therapy Center, "George Papanikolaou" Hospital, Thessaloniki, Greece.
Xing-Guo LiGraduate Institute of Biomedical Sciences, China Medical University, Taichung, Taiwan.
Pamela MeloHematopoietic Cell Transplantation Unit, Hematology Department, Gene and Cell Therapy Center, "George Papanikolaou" Hospital, Thessaloniki, Greece.
Monica VolpinSan Raffaele Telethon Institute for Gene Therapy-IRCCS Ospedale San Raffaele Scientific Institute, Milan, Italy.
Eugenio MontiniSan Raffaele Telethon Institute for Gene Therapy-IRCCS Ospedale San Raffaele Scientific Institute, Milan, Italy.
Mingdong LiuDepartment of Medicine, University of Washington, Seattle, Washington, USA.
Georgios KaltsounisHematopoietic Cell Transplantation Unit, Hematology Department, Gene and Cell Therapy Center, "George Papanikolaou" Hospital, Thessaloniki, Greece.ORCID 0000-0001-8803-9273
Minas YiangouDepartment of Genetics, Development and Molecular Biology, School of Biology, Aristotle University of Thessaloniki, Thessaloniki, Greece.
David W EmeryDepartment of Medicine, University of Washington, Seattle, Washington, USA.
Achilles AnagnostopoulosHematopoietic Cell Transplantation Unit, Hematology Department, Gene and Cell Therapy Center, "George Papanikolaou" Hospital, Thessaloniki, Greece.
Thalia PapayannopoulouDepartment of Medicine, University of Washington, Seattle, Washington, USA.
Suming HuangDepartment of Pediatrics, Pennsylvania State University College of Medicine, Hershey, Pennsylvania, USA.
George StamatoyannopoulosDepartment of Medicine, University of Washington, Seattle, Washington, USA.
Evangelia YannakiHematopoietic Cell Transplantation Unit, Hematology Department, Gene and Cell Therapy Center, "George Papanikolaou" Hospital, Thessaloniki, Greece.ORCID 0000-0002-9849-1279
University of Washington · USAristotle University of Thessaloniki · GRG. Papanikolaou General Hospital · GRIRCCS Ospedale San Raffaele · ITAltius Institute for Biomedical Sciences · USChina Medical University · TWPennsylvania State University · US

Funding

New Chromatin Insulators and Enhancers for Gene Therapy of the HemoglobinopathiesR01HL136375 · NHLBI · UNIVERSITY OF WASHINGTON · PI STAMATOYANNOPOULOS, THALIA · 2017 to 2020
$2.3M
NHLBI NIH HHS R01 HL136375
6 · The paper itself

Abstract

Despite the unequivocal success of hematopoietic stem and progenitor cell gene therapy, limitations still exist including genotoxicity and variegation/silencing of transgene expression. A class of DNA regulatory elements known as chromatin insulators (CIs) can mitigate both vector transcriptional silencing (barrier CIs) and vector-induced genotoxicity (enhancer-blocking CIs) and have been proposed as genetic modulators to minimize unwanted vector/genome interactions. Recently, a number of human, small-sized, and compact CIs bearing strong enhancer-blocking activity were identified. To ultimately uncover an ideal CI with a dual, enhancer-blocking and barrier activity, we interrogated these elements

Indexed as

ChromatinInsulator ElementsEnhancer Elements, GeneticGenetic TherapyGenetic VectorsHematopoietic Stem CellsHumansChromatinbarrier activitychromatin insulatorCTCF-binding sitegene therapyhematopoietic stem cells

Identifiers

PMID34477013
PMCPMC9206477
OpenAlexW3198354313

What Socratic holds

Textmetadata
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 Socratic graph.