ArticleMolecular therapy. Nucleic acids2023
Forced enhancer-promoter rewiring to alter gene expression in animal models.
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Article in Molecular therapy. Nucleic acids, 2023. 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
1.4field-weighted citation impact, top 20% of its field
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Who cites it
5 citing papers in PubMed, 9 citations in OpenAlex.
- Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.Blood advances · 2026Article
- Single-stranded DNA-binding proteins are essential components of the architectural LDB1 protein complex.Molecular cell · 2025Article
- Epigenetic Regulation of Erythropoiesis: From Developmental Programs to Therapeutic Targets.International journal of molecular sciences · 2025Review
- The cells are all-right: Regulation of the Lefty genes by separate enhancers in mouse embryonic stem cells.PLoS genetics · 2024Article
- Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation.Molecular therapy. Methods & clinical development · 2023Article
Corrections and comments
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Authors and funding
27 authors at 3 institutions in 4 countries.
Scott A PeslakDivision of Hematology/Oncology, Department of Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.
Selami DemirciCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Vemika ChandraDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Byoung RyuDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Saurabh K BhardwajDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Jing JiangDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Jeremy W RuponDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Robert E ThromDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Naoya UchidaCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Alexis LeonardCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Khaled EssawiCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Aylin C BonifacinoTranslational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA.
Allen E KrouseTranslational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA.
Nathaniel S LindeTranslational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD 20814, USA.
Robert E DonahueCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Francesca FerraraDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Matthew WielgoszDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Osheiza AbdulmalikDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Nicole HamagamiDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Paula Germino-WatnickCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Anh LeCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Rebecca ChuCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Malikiya HindsCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Mitchell J WeissDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.
Wei TongDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institutes (NHLBI), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Gerd A BlobelDivision of Hematology, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
National Institutes of Health · USChildren's Hospital of Philadelphia · USSt. Jude Children's Research Hospital · US
Funding
Retroviral Mediated Gene Transfer Into Primate Hematopoietic CellsZIAHL002338 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI HONG, SO GUN · 2019 to 2025
$26.3MRegulation of Hemoglobin SwitchingR01HL119479 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI Gerd A Blobel · 2013 to 2026
$8.0MHEMATOPOIESIS TRAINING GRANTT32DK007780 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI TONG, WEI · 1999 to 2023
$6.7MLnk Regulatory Functions in Hematopoietic Stem CellsR01HL095675 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI TONG, WEI · 2009 to 2021
$5.6MRegulation of Ribosome Biogenesis in Hematopoietic Stem CellsR01DK127738 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI TONG, WEI · 2020 to 2024
$3.0MNovel Regulation of Oncogenic NRAS Signaling in Myeloid MalignanciesR01CA271523 · NCI · CHILDREN'S HOSP OF PHILADELPHIA · PI Wei Tong · 2022 to 2026
$2.7MCellular Signaling Pathways in the Regulation of Fetal Hemoglobin for Treatment of Sickle Cell DiseaseK08DK129716 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI PESLAK, SCOTT ALAN · 2021 to 2025
$836kNCI NIH HHS R01 CA271523NHLBI NIH HHS R01 HL095675NHLBI NIH HHS R01 HL119479NIDDK NIH HHS K08 DK129716NIDDK NIH HHS R01 DK127738NIDDK NIH HHS T32 DK007780
Abstract
Transcriptional enhancers can be in physical proximity of their target genes via chromatin looping. The enhancer at the β-globin locus (locus control region [LCR]) contacts the fetal-type (
Indexed as
enhancer-promoter interactionsfetal hemoglobinforced chromatin loopinghemoglobin switchingMT: Oligonucleotidespreclinical animal modelssickle cell diseaseTherapies and Applicationsvector optimization
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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.