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Minireview | Spotlight

Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies

Vigneshwaran Venkatesan, Saranya Srinivasan, Prathibha Babu, Saravanabhavan Thangavel
Vigneshwaran Venkatesan
aCentre for Stem Cell Research (CSCR), InStem Bengaluru, Christian Medical College, Vellore, Tamil Nadu, India
bManipal Academy of Higher Education, Manipal, Karnataka, India
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Saranya Srinivasan
aCentre for Stem Cell Research (CSCR), InStem Bengaluru, Christian Medical College, Vellore, Tamil Nadu, India
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Prathibha Babu
aCentre for Stem Cell Research (CSCR), InStem Bengaluru, Christian Medical College, Vellore, Tamil Nadu, India
bManipal Academy of Higher Education, Manipal, Karnataka, India
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Saravanabhavan Thangavel
aCentre for Stem Cell Research (CSCR), InStem Bengaluru, Christian Medical College, Vellore, Tamil Nadu, India
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  • ORCID record for Saravanabhavan Thangavel
DOI: 10.1128/MCB.00253-20
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ABSTRACT

β-Hemoglobinopathies are the most common monogenic disorders, and a century of research has provided us with a better understanding of the attributes of these diseases. Allogenic stem cell transplantation was the only potentially curative option available for these diseases until the discovery of gene therapy. The findings on the protective nature of fetal hemoglobin in sickle cell disease (SCD) and thalassemia patients carrying hereditary persistence of fetal hemoglobin (HPFH) mutations has given us the best evidence that the cure for β-hemoglobinopathies remains hidden in the hemoglobin locus. The detailed understanding of the developmental gene regulation of gamma-globin (γ-globin) and the emergence of gene manipulation strategies offer us the opportunity for developing a γ-globin gene-modified autologous stem cell transplantation therapy. In this review, we summarize different therapeutic strategies that reactivate fetal hemoglobin for the gene therapy of β-hemoglobinopathies.

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Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies
Vigneshwaran Venkatesan, Saranya Srinivasan, Prathibha Babu, Saravanabhavan Thangavel
Molecular and Cellular Biology Dec 2020, 41 (1) e00253-20; DOI: 10.1128/MCB.00253-20

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Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies
Vigneshwaran Venkatesan, Saranya Srinivasan, Prathibha Babu, Saravanabhavan Thangavel
Molecular and Cellular Biology Dec 2020, 41 (1) e00253-20; DOI: 10.1128/MCB.00253-20
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  • Top
  • Article
    • ABSTRACT
    • INTRODUCTION
    • β-HEMOGLOBINOPATHIES AND THEIR PATHOPHYSIOLOGY
    • HEMOGLOBIN SWITCHING
    • BENEFICIAL MUTATIONS ALTERING DEVELOPMENTALLY REGULATED EXPRESSION
    • THERAPEUTIC STRATEGIES FOR HbF REACTIVATION
    • CONCLUSION
    • ACKNOWLEDGMENTS
    • REFERENCES
  • Figures & Data
  • Info & Metrics
  • PDF

KEYWORDS

hemoglobinopathies
gene editing
hematopoietic stem cells

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