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Rivella Laboratory Publications

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Researchers in the Rivella Lab publish extensively. See notable highlights of their work can be found below.

Total:
25

2024

Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia
American Journal Of Hematology · July, 2024
Use of HSC Targeted LNP to Generate a Mouse Model of Lethal α-Thalassemia and Treatment via Lentiviral Gene Therapy
Blood · July, 2024

2023

In vivo hematopoietic stem cell modification by mRNA delivery
Science (New York, N.Y.) · July, 2023

2021

Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin
Haematologica · October, 2021
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies
Molecular Therapy : The Journal Of The American Society Of Gene Therapy · April, 2021

2020

Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activity
Blood · October, 2020
Minihepcidins improve ineffective erythropoiesis and splenomegaly in a new mouse model of adult β-thalassemia major
Haematologica · July, 2020

2019

Lobe specificity of iron binding to transferrin modulates murine erythropoiesis and iron homeostasis
Blood · October, 2019
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance
Haematologica · May, 2019
Iron metabolism under conditions of ineffective erythropoiesis in β-thalassemia
Blood · January, 2019

2018

Ineffective Erythropoiesis: Anemia and Iron Overload
Hematology/oncology Clinics Of North America · April, 2018
Short-term administration of JAK2 inhibitors reduces splenomegaly in mouse models of β-thalassemia intermedia and major
Haematologica · February, 2018

2017

A Red Carpet for Iron Metabolism
Cell · January, 2017

2016

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers
Blood · August, 2016
Minihepcidin peptides as disease modifiers in mice affected by β-thalassemia and polycythemia vera
Blood · July, 2016

2014

Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine β-thalassemia
Blood · June, 2014
Distinct roles for hepcidin and interleukin-6 in the recovery from anemia in mice injected with heat-killed Brucella abortus
Blood · February, 2014

2013

Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice
The Journal Of Clinical Investigation · April, 2013
Macrophages support pathological erythropoiesis in polycythemia vera and β-thalassemia
Nature Medicine · April, 2013

2010

Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice
The Journal Of Clinical Investigation · December, 2010

2008

Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia
Blood · August, 2008

2007

Ineffective erythropoiesis in beta-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin
Blood · June, 2007

2003

A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human beta-globin gene transfer
Blood · April, 2003

2000

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin
Nature · July, 2000
The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites
Journal Of Virology · May, 2000