• Users Online: 52
  • Print this page
  • Email this page
REVIEW ARTICLE
Year : 2020  |  Volume : 3  |  Issue : 3  |  Page : 146-154

Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution


King Abdullah International Medical Research Center, Stem Cells Unit; Department of Cellular Therapy and Cancer Research, King Saud Bin Abdulaziz University for Health Sciences, Riyadh, Saudi Arabia

Correspondence Address:
Bahauddeen M Alrfaei
Stem Cells and Regenerative Medicine Unit, Department of Cellular Therapy and Cancer Research, King Abdullah International Medical Research Center, Riyadh
Saudi Arabia
Login to access the Email id

Source of Support: None, Conflict of Interest: None


DOI: 10.4103/JNSM.JNSM_59_19

Rights and Permissions

Hemoglobin (Hb) disorders or hemoglobinopathies are groups of blood conditions involving inherited genetic diseases – mostly as single-gene autosomal recessive – that lead to the formation of abnormal Hb structure or inadequate to no production of globin chains in Hbs. Disorders of Hb are a global concern since these diseases can cause severe morbidity and early mortality of the affected populations. Treatments vary between chemicals and molecular approaches. The most promising approach is Hb correction. However, the stability of the correction faces a big challenge along with safety concerns. It is worth noting that most of the inherited hemoglobinopathies share common clinical presentations and laboratory findings, although some have distinct features. Hemoglobinopathies with emphasis on recent advances in gene therapy targeting sickle cell disease and thalassemia are discussed in this review.


[FULL TEXT] [PDF]*
Print this article     Email this article
 Next article
 Previous article
 Table of Contents

 Similar in PUBMED
   Search Pubmed for
   Search in Google Scholar for
 Related articles
 Citation Manager
 Access Statistics
 Reader Comments
 Email Alert *
 Add to My List *
 * Requires registration (Free)
 

 Article Access Statistics
    Viewed729    
    Printed68    
    Emailed0    
    PDF Downloaded150    
    Comments [Add]    

Recommend this journal