Tmprss6-ASO as a tool for the treatment of Polycythemia Vera mice

Carla Casu; Alison Liu; Gianluca De Rosa; Audrey Low; Aae Suzuki; Sayantani Sinha; Yelena Z Ginzburg; Charles Abrams; Mariam Aghajan; Shuling Guo; Stefano Rivella

doi:10.1371/journal.pone.0251995

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Tmprss6-ASO as a tool for the treatment of Polycythemia Vera mice

Journal article

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Tmprss6-ASO as a tool for the treatment of Polycythemia Vera mice

Carla Casu, Alison Liu, Gianluca De Rosa, Audrey Low, Aae Suzuki, Sayantani Sinha, Yelena Z Ginzburg, Charles Abrams, Mariam Aghajan, Shuling Guo, …

PloS one, v 16(12), e0251995

10 Dec 2021

DOI: https://doi.org/10.1371/journal.pone.0251995

PMID: 34890402

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Abstract

Animals

Humans

Janus Kinase 2 - genetics

Janus Kinase 2 - metabolism

Membrane Proteins - antagonists & inhibitors

Membrane Proteins - genetics

Membrane Proteins - metabolism

Mice, Transgenic

Oligonucleotides, Antisense - genetics

Oligonucleotides, Antisense - pharmacology

Polycythemia Vera - drug therapy

Polycythemia Vera - genetics

Polycythemia Vera - metabolism

RNA, Messenger - antagonists & inhibitors

RNA, Messenger - genetics

RNA, Messenger - metabolism

Serine Endopeptidases - genetics

Serine Endopeptidases - metabolism

Polycythemia Vera (PV) is a chronic myeloproliferative neoplasm resulting from an acquired driver mutation in the JAK2 gene of hematopoietic stem and progenitor cells resulting in the overproduction of mature erythrocytes and abnormally high hematocrit, in turn leading to thromboembolic complications. Therapeutic phlebotomy is the most common treatment to reduce the hematocrit levels and consequently decrease thromboembolic risk. Here we demonstrate that, by using the iron restrictive properties of the antisense oligonucleotides against Tmprss6 mRNA, we can increase hepcidin to achieve effects equivalent to therapeutic phlebotomy. We provide evidence that this less invasive approach could represent an additional therapeutic tool for the treatment of PV patients.

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Title: Tmprss6-ASO as a tool for the treatment of Polycythemia Vera mice
Creators: Carla Casu - Children's Hospital of Philadelphia
Alison Liu - Children's Hospital of Philadelphia
Gianluca De Rosa - Children's Hospital of Philadelphia
Audrey Low - Ionis Pharmaceuticals
Aae Suzuki - University of Pennsylvania
Sayantani Sinha - Children's Hospital of Philadelphia
Yelena Z Ginzburg - Icahn School of Medicine at Mount Sinai
Charles Abrams - University of Pennsylvania
Mariam Aghajan - Ionis Pharmaceuticals
Shuling Guo - Ionis Pharmaceuticals
Stefano Rivella - Children's Hospital of Philadelphia
Publication Details: PloS one, v 16(12), e0251995
Publisher: PLOS
Grant note: P01 HL146373 / NHLBI NIH HHS R01 DK090554 / NIDDK NIH HHS R01 HL146722 / NHLBI NIH HHS R01 DK095112 / NIDDK NIH HHS
Resource Type: Journal article
Language: English
Academic Unit: Pharmacology and Physiology
Web of Science ID: WOS:000747293600001
Scopus ID: 2-s2.0-85121024341
Other Identifier: 991022004871204721

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Collaboration types: Industry collaboration; Domestic collaboration
Web of Science research areas: Hematology

Tmprss6-ASO as a tool for the treatment of Polycythemia Vera mice

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Abstract

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Details

UN Sustainable Development Goals (SDGs)

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