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Background <p>β-Thalassemia and sickle cell disease are the most common hereditary β-hemoglobinopathies.&#xa0;The current available treatments for their severe cases include frequent blood transfusions, iron chelation therapy, and allogenic hematopoietic stem cell transplantation (HSCT). Clustered Regularly Interspaced Short Palindromic Repeats (Crispr), offer a potentially safe and effective cure for these patients. This systematic review aims to report on its value in treating transfusion-dependent β-thalassemia (TDT), and sickle cell disease.</p> Methods <p>This systematic review was conducted according to the Preferred Reporting Items of Systematic Reviews and Meta-analyses (PRISMA). A comprehensive search was done across available databases (PubMed, Scopus, Web of Science), until October 2024. Clinical trials on transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) patients undergoing Crispr/Cas9 gene editing technology, were included in this systematic review. Relevant data was extracted and interpreted.</p> Results <p>Four non-randomized single-arm clinical trials, reporting a total of 101 patients in the full-analysis population, with a mean age of 18.1 years, were included in this review. Positive results were observed where TDT patients achieved transfusion independence, and vaso-occlusive events were either reduced or absent in SCD patients, across the follow-up duration. Notably, positive fetal hemoglobin (HbF) was achieved, aiding in the chronic anemia correction. Grade ≥ 3 adverse events were reported in 87.1% of patients, most commonly febrile neutropenia.</p> Conclusion <p>Crispr/Cas9 reported high efficacy, with transfusion-independence and reducing vaso-occlusive events. HbF levels were significantly raised correcting the associated chronic anemia and elevating quality of life. Unfortunately, the incidence of grade ≥ 3 adverse events was high in the treated population, raising concerns about the potential safety of this technology.</p>

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Crispr/Cas9 gene editing in transfusion-dependent β-thalassemia and sickle cell disease: a systematic review of non-randomized clinical trials

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年11月10日

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  • Basma M. El-Khalifa,
  • Weaam A. Soliman,
  • Hazem M. Mazy,
  • Muhtadi G. Ahmed,
  • Hamza Al-Trad,
  • Tarek H. Elmeneizee,
  • Hossam W. Abd El-Aziz,
  • Selma Ahmed Hassan Abdelmutaleb,
  • Ghada A. Abdelghaffar,
  • Maha H. Morsi

摘要

Background

β-Thalassemia and sickle cell disease are the most common hereditary β-hemoglobinopathies. The current available treatments for their severe cases include frequent blood transfusions, iron chelation therapy, and allogenic hematopoietic stem cell transplantation (HSCT). Clustered Regularly Interspaced Short Palindromic Repeats (Crispr), offer a potentially safe and effective cure for these patients. This systematic review aims to report on its value in treating transfusion-dependent β-thalassemia (TDT), and sickle cell disease.

Methods

This systematic review was conducted according to the Preferred Reporting Items of Systematic Reviews and Meta-analyses (PRISMA). A comprehensive search was done across available databases (PubMed, Scopus, Web of Science), until October 2024. Clinical trials on transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) patients undergoing Crispr/Cas9 gene editing technology, were included in this systematic review. Relevant data was extracted and interpreted.

Results

Four non-randomized single-arm clinical trials, reporting a total of 101 patients in the full-analysis population, with a mean age of 18.1 years, were included in this review. Positive results were observed where TDT patients achieved transfusion independence, and vaso-occlusive events were either reduced or absent in SCD patients, across the follow-up duration. Notably, positive fetal hemoglobin (HbF) was achieved, aiding in the chronic anemia correction. Grade ≥ 3 adverse events were reported in 87.1% of patients, most commonly febrile neutropenia.

Conclusion

Crispr/Cas9 reported high efficacy, with transfusion-independence and reducing vaso-occlusive events. HbF levels were significantly raised correcting the associated chronic anemia and elevating quality of life. Unfortunately, the incidence of grade ≥ 3 adverse events was high in the treated population, raising concerns about the potential safety of this technology.