Article

Gene Therapy of Hemoglobinopathies

2025-11-13

Abstract excerpt

<h4>Background: </h4> /Objectives: Sickle cells disease (SCD) and -thalassemia are autosomal recessive disorders of erythroid cells due to gene mutations occurring at the level of the -globin gene. The severe forms of these hemoglobinopathies observed in individuals homozygous for these defective genes need intensive treatments, are associated with a poor quality of life and allogeneic hematopoietic stem cell repr...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 475a822b-8984-5e70-8417-718c34b6ce00
DOI: 10.20944/preprints202511.0971.v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Gene Therapy of HemoglobinopathiesDOI 10.20944/preprints202511.0971.v1Read

Gene therapy for sickle cell disease and thalassemiaSemantic proximity Read

A Review of Gene Therapies for HemoglobinopathiesSemantic proximity Read

The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challengesSemantic proximity Read

Recent advancements in gene therapy for sickle cell disease and β-thalassemiaSemantic proximity Read

Gene Therapy for HemoglobinopathiesSemantic proximity Read

Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative MedicineSemantic proximity Read

Advances in Gene Therapy for Inherited HaemoglobinopathiesSemantic proximity Read

Gene Therapy of the HemoglobinopathiesSemantic proximity Read

Promise of gene therapy to treat sickle cell diseaseSemantic proximity Read

Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.Semantic proximity Read

Gene therapy of hemoglobinopathies: progress and future challengesSemantic proximity Read

Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene EditingSemantic proximity Read