ArticleTrends in molecular medicine2025
The bleeding edge: broadening horizons for hematopoietic stem cell therapies.
Spencer D Shelton, Vijay G Sankaran
Abstract read
In one paragraphArticle in Trends in molecular medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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0citing papers in PubMed
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What it found
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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 registryThe 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.
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3 · Its place in the literatureWho cites it
0 citing papers in PubMed.
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4 · The recordCorrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
5 · Who and what moneyAuthors and funding
2 authors.
Spencer D SheltonDivision of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA; Howard Hughes Medical Institute, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Vijay G SankaranDivision of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA 02215, USA; Howard Hughes Medical Institute, Boston, MA 02115, USA; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA. Electronic address: sankaran@broadinstitute.org.
Funding
TRANSFUSION BIOLOGY AND MEDICINET32HL066987 · NHLBI · CHILDREN'S HOSPITAL BOSTON · PI JOHN P MANIS · 2001 to 2026
$10.2MSystematic Genetic Dissection of Human ErythropoiesisR01DK103794 · NIDDK · BOSTON CHILDREN'S HOSPITAL · PI Vijay Ganesh Sankaran · 2014 to 2026
$5.9MNext generation functional genomics of hematology traitsR01HL146500 · NHLBI · UNIVERSITY OF WASHINGTON · PI ALEXANDER P REINER · 2020 to 2026
$5.7MSelective pressures from inherited variation impacting myeloproliferative neoplasm initiationR01CA265726 · NCI · BOSTON CHILDREN'S HOSPITAL · PI Vijay Ganesh Sankaran · 2022 to 2026
$2.5MVariant to Function Mapping of B-ALL Risk LociR01CA292941 · NCI · BOSTON CHILDREN'S HOSPITAL · PI Adam De Smith, Vijay Ganesh Sankaran · 2024 to 2026
$2.0MClonal analysis of cancer by mitochondrial DNA barcodingR33CA278393 · NCI · BRIGHAM AND WOMEN'S HOSPITAL · PI SANKARAN, VIJAY GANESH, VAN GALEN, PETER · 2023 to 2025
$1.3MNCI NIH HHS R01 CA265726NCI NIH HHS R01 CA292941NCI NIH HHS R33 CA278393NHLBI NIH HHS R01 HL146500NHLBI NIH HHS T32 HL066987NIDDK NIH HHS R01 DK103794
6 · The paper itselfAbstract
Recent reports of gene therapy using autologous hematopoietic stem cell transplantation (HSCT) have addressed protein deficiencies of extra-hematopoietic origin. In a recent study, Srivastava et al. report that patients with hemophilia A receiving F8 lentiviral HSCT gene therapy achieved lasting factor VIII restoration and clinical improvement, marking an advance that could enable broader applications of HSCT.
Indexed as
Genetic TherapyHematopoietic Stem Cell TransplantationHemophilia AHemorrhageAnimalsFactor VIIIHematopoietic Stem CellsHumansLentivirusFactor VIIIgene therapyhematopoiesishematopoietic stem cellhemophilialineage-specific
Identifiers
PMID40121135
PMCPMC11985263
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