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Medicine (Baltimore) ; 103(18): e38036, 2024 May 03.
Artigo em Inglês | MEDLINE | ID: mdl-38701251

RESUMO

ß-Thalassemia is the world's number 1 single-gene genetic disorder and is characterized by suppressed or impaired production of ß-pearl protein chains. This results in intramedullary destruction and premature lysis of red blood cells in peripheral blood. Among them, patients with transfusion-dependent ß-thalassemia face the problem of long-term transfusion and iron chelation therapy, which leads to clinical complications and great economic stress. As gene editing technology improves, we are seeing the dawn of a cure for the disease, with its reduction of ineffective erythropoiesis and effective prolongation of survival in critically ill patients. Here, we provide an overview of ß-thalassemia distribution and pathophysiology. In addition, we focus on gene therapy and gene editing advances. Nucleic acid endonuclease tools currently available for gene editing fall into 3 categories: zinc finger nucleases, transcription activator-like effector nucleases, and regularly interspaced short palindromic repeats (CRISPR-Cas9) nucleases. This paper reviews the exploratory applications and exploration of emerging therapeutic tools based on 3 classes of nucleic acid endonucleases in the treatment of ß-thalassemia diseases.


Assuntos
Edição de Genes , Terapia Genética , Talassemia beta , Talassemia beta/terapia , Talassemia beta/genética , Humanos , Edição de Genes/métodos , Terapia Genética/métodos , Sistemas CRISPR-Cas , Nucleases dos Efetores Semelhantes a Ativadores de Transcrição/genética , Nucleases de Dedos de Zinco/genética
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