Generation of an in vitro model of β‐thalassemia using the CRISPR/Cas9 genome editing system

Short-hairpin RNA against aberrant HBB[IVSI-110(G>A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

(2018) Petros Patsali et al.   HAEMATOLOGICA

Short-hairpin RNA against aberrant HBB[IVSI-110(G>A)] mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells

(2018) Petros Patsali et al.   HAEMATOLOGICA

Combination therapy with aTmprss6RNAi-therapeutic and the oral iron chelator deferiprone additively diminishes secondary iron overload in a mouse model of β-thalassemia intermedia

(2015) Paul J. Schmidt et al.   AMERICAN JOURNAL OF HEMATOLOGY

EHMT1 and EHMT2 inhibition induces fetal hemoglobin expression

(2015) A. Renneville et al.   BLOOD

Erythroid differentiation ability of butyric acid analogues: Identification of basal chemical structures of new inducers of foetal haemoglobin

(2015) Nicoletta Bianchi et al.   EUROPEAN JOURNAL OF PHARMACOLOGY

 -thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies

(2015) S. Rivella   HAEMATOLOGICA

A mouse model to study thrombotic complications of thalassemia

(2015) Yosef Kalish et al.   THROMBOSIS RESEARCH

 -thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies

(2015) S. Rivella   HAEMATOLOGICA

Modified activin receptor IIB ligand trap mitigates ineffective erythropoiesis and disease complications in murine  -thalassemia

(2014) R. N. V. S. Suragani et al.   BLOOD

Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects

(2014) Bin Shen et al.   NATURE METHODS

Quantitative analysis of murine terminal erythroid differentiation in vivo: novel method to study normal and disordered erythropoiesis

(2013) J. Liu et al.   BLOOD

Genome engineering using the CRISPR-Cas9 system

(2013) F Ann Ran et al.   Nature Protocols

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients

(2012) Laura Breda et al.   PLoS One

 -thalassemia: a model for elucidating the dynamic regulation of ineffective erythropoiesis and iron metabolism

(2011) Y. Ginzburg et al.   BLOOD

Transferrin therapy ameliorates disease in β-thalassemic mice

(2010) Huihui Li et al.   NATURE MEDICINE

The 3′ Region of the Chicken Hypersensitive Site-4 Insulator Has Properties Similar to Its Core and Is Required for Full Insulator Activity

(2009) Paritha I. Arumugam et al.   PLoS One

Differentiation of Embryonic Stem Cells to Clinically Relevant Populations: Lessons from Embryonic Development

(2008) Charles E. Murry et al.   CELL