Journal of Nature and Science of Medicine

REVIEW ARTICLE
Year
: 2020  |  Volume : 3  |  Issue : 3  |  Page : 146--154

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


Bader Al Alwan, Arwa A Alsubait, Bahauddeen M Alrfaei 
 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

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.


How to cite this article:
Al Alwan B, Alsubait AA, Alrfaei BM. Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution.J Nat Sci Med 2020;3:146-154


How to cite this URL:
Al Alwan B, Alsubait AA, Alrfaei BM. Hemoglobinopathy correction with CRISPR or not; gene therapy is the solution. J Nat Sci Med [serial online] 2020 [cited 2020 Sep 25 ];3:146-154
Available from: http://www.jnsmonline.org/article.asp?issn=2589-627X;year=2020;volume=3;issue=3;spage=146;epage=154;aulast=Al;type=0