Topic: Thalassemia: Progress in Research and Insights

Thalassemia is a group of genetic blood disorders characterized by abnormal hemoglobin production, which leads to anemia. Thalassemia affects individuals of all ages and ethnicities, with severity ranging from mild symptoms requiring minimal intervention to severe forms necessitating regular blood transfusions and iron chelation therapy.

Current treatment options for thalassemia include blood transfusions to alleviate anemia and iron chelation therapy to manage the iron overload resulting from frequent transfusions. However, these treatments are palliative and do not address the root cause of the disorder. Gene therapy has emerged as a promising approach with the potential to cure thalassemia by correcting the genetic mutations responsible for abnormal hemoglobin production.

This Special Issue on Thalassemia will focus on the latest advancements in gene therapy, including next-generation gene editing technologies aimed at correcting the genetic defects underlying the disorder. Additionally, we will explore novel approaches to enhancing the efficiency of gene delivery to hematopoietic stem cells, optimizing therapeutic cargo expression, and improving patient outcomes.

We welcome Original Research Articles, Review Articles, and Brief Communications on topics that include but are not limited to:
- Gene editing tools such as CRISPR/Cas9 for thalassemia treatment;
- Regulations for clinical trials of gene therapy in thalassemia;
- Patient advocacy and involvement in research initiatives;
- Innovative strategies for non-viral gene delivery to hematopoietic cells.

Join us as we delve into the exciting developments that are shaping the future of thalassemia treatment and care.

15 Jan 2025