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IthaGenes: an interactive database for haemoglobin variations and epidemiology

PloS one, 2014-07, Vol.9 (7), p.e103020-e103020

2014

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Acid Ceramidase Depletion Impairs Neuronal Survival and Induces Morphological Defects in Neurites Associated with Altered Gene Transcription and Sphingolipid Content

International journal of molecular sciences, 2020-02, Vol.21 (5), p.1607

2020

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Therapy Development by Genome Editing of Hematopoietic Stem Cells

Cells (Basel, Switzerland), 2021-06, Vol.10 (6), p.1492

2021

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Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells

International journal of molecular sciences, 2021-03, Vol.22 (7), p.3626

2021

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CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation

International journal of molecular sciences, 2022-01, Vol.23 (3), p.1082

2022

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Pathways and genes differentially expressed in the motor cortex of patients with sporadic amyotrophic lateral sclerosis

BMC genomics, 2007-01, Vol.8 (1), p.26-26, Article 26

2007

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Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBBIVSI−110 (G > A) β-Thalassemia

International journal of molecular sciences, 2020-09, Vol.21 (18), p.6671

2020

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The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34 + Cells from Sickle Cell Disease Patients

Viruses, 2022-12, Vol.14 (12), p.2716

2022

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Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

Pharmaceutics, 2022-04, Vol.14 (4), p.793

2022

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Selection and Identification of Skeletal-Muscle-Targeted RNA Aptamers

Molecular therapy. Nucleic acids, 2018-03, Vol.10, p.199-214

2018

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Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference

Cells (Basel, Switzerland), 2023-12, Vol.12 (24), p.2848

2023

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High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis

Frontiers in genome editing, 2023-03, Vol.5, p.1141618-1141618

2023

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Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

Molecular diagnosis & therapy, 2019-04, Vol.23 (2), p.201-222

2019

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Disruptive Technology: CRISPR/Cas-Based Tools and Approaches

Molecular diagnosis & therapy, 2019-04, Vol.23 (2), p.187-200

2019

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novel family of katanin-like 2 protein isoforms (KATNAL2), interacting with nucleotide-binding proteins Nubp1 and Nubp2, are key regulators of different MT-based processes in mammalian cells

Cellular and molecular life sciences : CMLS, 2016-01, Vol.73 (1), p.163-184

2016

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Differential expression of molecular motors in the motor cortex of sporadic ALS

Neurobiology of disease, 2007-06, Vol.26 (3), p.577-589

2007

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Adapting the ACMG/AMP variant classification framework: A perspective from the ClinGen Hemoglobinopathy Variant Curation Expert Panel

Human mutation, 2022-08, Vol.43 (8), p.1089-1096

2022

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TMEM147 interacts with lamin B receptor, regulates its localization and levels, and affects cholesterol homeostasis

Journal of cell science, 2020-08, Vol.133 (16)

2020

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57Fe enrichment in mice for β-thalassaemia studies via Mössbauer spectroscopy of blood samples

European biophysics journal, 2019-10, Vol.48 (7), p.635-643

2019

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Context base editing for splice correction of IVSI-110 β-thalassemia

Molecular therapy. Nucleic acids, 2024-06, Vol.35 (2), p.102183-102183, Article 102183

2024

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The molecular spectrum and distribution of haemoglobinopathies in Cyprus: a 20-year retrospective study

Scientific reports, 2016-05, Vol.6 (1), p.26371-26371, Article 26371

2016

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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

Haematologica (Roma), 2018-09, Vol.103 (9), p.e419

2018

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A Novel BaEVRless-Pseudotyped γ-Globin Lentiviral Vector Drives High and Stable Fetal Hemoglobin Expression and Improves Thalassemic Erythropoiesis In Vitro

Human gene therapy, 2019-05, Vol.30 (5), p.601

2019

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Recent trends in the gene therapy of β-thalassemia

Journal of Blood Medicine, 2015-01, Vol.6 (default), p.69-85

2015

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nucleotide-binding proteins Nubp1 and Nubp2 are negative regulators of ciliogenesis

Cellular and molecular life sciences : CMLS, 2014-02, Vol.71 (3), p.517-538

2014

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IthaGenes: an interactive database for haemoglobin variations and epidemiology

Acid Ceramidase Depletion Impairs Neuronal Survival and Induces Morphological Defects in Neurites Associated with Altered Gene Transcription and Sphingolipid Content

Therapy Development by Genome Editing of Hematopoietic Stem Cells

Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells

CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation

Pathways and genes differentially expressed in the motor cortex of patients with sporadic amyotrophic lateral sclerosis

Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBBIVSI−110 (G > A) β-Thalassemia

The Optimized γ-Globin Lentiviral Vector GGHI-mB-3D Leads to Nearly Therapeutic HbF Levels In Vitro in CD34 + Cells from Sickle Cell Disease Patients

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

Selection and Identification of Skeletal-Muscle-Targeted RNA Aptamers

Evaluation of Mono- and Bi-Functional GLOBE-Based Vectors for Therapy of β-Thalassemia by HBBAS3 Gene Addition and Mutation-Specific RNA Interference

High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

Disruptive Technology: CRISPR/Cas-Based Tools and Approaches

novel family of katanin-like 2 protein isoforms (KATNAL2), interacting with nucleotide-binding proteins Nubp1 and Nubp2, are key regulators of different MT-based processes in mammalian cells

Differential expression of molecular motors in the motor cortex of sporadic ALS

Adapting the ACMG/AMP variant classification framework: A perspective from the ClinGen Hemoglobinopathy Variant Curation Expert Panel

TMEM147 interacts with lamin B receptor, regulates its localization and levels, and affects cholesterol homeostasis

57Fe enrichment in mice for β-thalassaemia studies via Mössbauer spectroscopy of blood samples

Context base editing for splice correction of IVSI-110 β-thalassemia

The molecular spectrum and distribution of haemoglobinopathies in Cyprus: a 20-year retrospective study

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

A Novel BaEVRless-Pseudotyped γ-Globin Lentiviral Vector Drives High and Stable Fetal Hemoglobin Expression and Improves Thalassemic Erythropoiesis In Vitro

Recent trends in the gene therapy of β-thalassemia

nucleotide-binding proteins Nubp1 and Nubp2 are negative regulators of ciliogenesis

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