Regulation of gene expression in hematopoiesis
Author(s):
Mohsin Badat
Affiliations:
MRC Weatherall Institute of Molecular Medicine, University of Oxford, United Kingdom
EHA Library. Higgs D. Jun 15, 2018; 219085 Topic: 8Aa Hematopoiesis and stem cell biology
Prof. Dr. Douglas Higgs
Prof. Dr. Douglas Higgs
Contributions
Learning Objectives
THIS MANUSCRIPT IS PUBLISHED AS AN OFFICIAL SUPPLEMENT OF HEMASPHERE.

Maria Domenica Cappellini - Chair Introduction
The survival and quality of life (QoL) of patients with hemoglobin disorder in developed countries have improved markedly in recent decades. Despite important improvements in the management of thalassemias, and sickle cell disease (SCD) there are still many challenges to overcome before global disease control is achievable. Our understanding of the underlying pathophysiological mechanisms of thalassemias and SCD and their associated clinical morbidity has increased substantially however there is an increasing awareness of the limitations of current management strategies. All these are driving research into novel therapeutic options for this patient population. In this session Dr. Doug Higgs, will illustrates how the analysis of the globin genes switching has pioneered the application of genetic engineering to hemoglobinopathies which are amongst the first examples of successful gene therapy, and they provide ideal targets for current developments in therapeutic genome editing. Dr. Brigitte Ranque will point out that the African setting has many particularities including a high infectious burden, very poor socio-economic conditions and underdeveloped health care system, impacting on a shorten life expectancy for SC patients in sub-Saharan Africa, compared to United States. She will present the CADRE study (Coeur Artères et DREpanocytose, i.e. Heart Arteries and Sickle cell disease) which is the world largest ongoing cohort of SCD and she presents the challenges of an Africa multinational study on SCD. Dr. Miguel Abboud will update on ongoing clinical trials in SCD and Thalassemia. He will focus on different targets for SCD: HbF induction, inhibition of sickle polymerization, inhibition of adhesion and cell-cell interactions, anticoagulants and anti-platelet agents, nitric oxide production and antioxidants and for thalassemia he will focus on agents targeting ineffective erythropoiesis.

Learning Objectives of the article
- To understand the new opportunities to regulate gene switch/expression in human genetic disease.
- To know the history and prognosis factors of SCD patients living in sub-Saharan Africa.
- To be updated on new therapeutic approaches for hemoglobinopathies.

Learning Objectives of the presentation
After viewing this presentation the participant will be able to:
- Describe in outline the current models underlying regulation of mammalian gene expression
- Understand the principles of genome editing
- Have an overview of how genome editing of hematopoietic stem cells could be used for therapy

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