Applications and research advances in the delivery of CRISPR/Cas9 systems for the treatment of inherited diseases
X Lu, M Zhang, G Li, S Zhang, J Zhang, X Fu… - International journal of …, 2023 - mdpi.com
The rapid advancements in gene therapy have opened up new possibilities for treating
genetic disorders, including Duchenne muscular dystrophy, thalassemia, cystic fibrosis …
genetic disorders, including Duchenne muscular dystrophy, thalassemia, cystic fibrosis …
Global, regional, and national burden of thalassemia, 1990–2021: a systematic analysis for the global burden of disease study 2021
Y Tuo, Y Li, Y Li, J Ma, X Yang, S Wu, J Jin, Z He - Eclinicalmedicine, 2024 - thelancet.com
Background Anemia is a significant contributor to the global disease burden, of which
thalassemia is the most common hereditary anaemic disease. Previous estimates were …
thalassemia is the most common hereditary anaemic disease. Previous estimates were …
Therapeutic Relevance of Inducing Autophagy in β-Thalassemia
The β-thalassemias are inherited genetic disorders affecting the hematopoietic system. In β-
thalassemias, more than 350 mutations of the adult β-globin gene cause the low or absent …
thalassemias, more than 350 mutations of the adult β-globin gene cause the low or absent …
Pharmacogenomics of Drugs Used in β-Thalassemia and Sickle-Cell Disease: From Basic Research to Clinical Applications
In this short review we have presented and discussed studies on pharmacogenomics (also
termed pharmacogenetics) of the drugs employed in the treatment of β-thalassemia or Sickle …
termed pharmacogenetics) of the drugs employed in the treatment of β-thalassemia or Sickle …