Journal

ACE Journal of Research Studies in Biosciences

  • ISSN Print: 2520-3916, ISSN Online:
  • Start Year: 2021
  • Volume: 1 (2021), Issue: 3

Gene Therapy Targeting Hematopoietic Stem Cells for ??Thalassemia: Challenge and Progress: A Review

Author: Rawaa Abdul?Ameer Abdul?Jabbar, Seeham Ali Qasim and Ghanyia Jasim Shanyoor
Page: 20-27
Published online: 16 Aug 2021

https://doi.org/

Abstract:

In general β?thalassemia is a disorder in the blood due to failure in production and synthesis of β?globin chains that leads to sickness and mostly ends with mortality because of low levels of hemoglobin. In this review article, the research went through an introduction gene therapy and how the editing of genes helps patients to recover from the diseases through gene therapy that was firstly discovered in 1945 and confirmed in 1960 and the first trials were carried out between 1970’s and 1980’s. However, the most important trials and successful achievements were started from 1999 until now. Implementation of gene therapy facing challenges such as high cost, the techniques need to be developed, the gene holders (viral) and the safety.