Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

Chimeric antigen receptor (CAR) T-cell therapy has proven a breakthrough in cancer
treatment in the last decade, giving unprecedented results against hematological …

Precise genome editing is crucial for establishing isogenic human disease models and ex
vivo stem cell therapy from the patient-derived hPSCs. Unlike Cas9-mediated knock-in …

β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …

Cardiovascular diseases are the leading cause of morbidity and mortality worldwide,
highlighting the high socioeconomic impact. Current treatment strategies like compound …

One of the most recent advances in the genome editing field has been the addition of “TALE
Base Editors”, an innovative platform for cell therapy that relies on the deamination of …

Abstract Beta-thalassemia (β-thalassemia) is an autosomal recessive disorder caused by
point mutations, insertions, and deletions in the HBB gene cluster, resulting in the …

摘要地中海贫血(地贫) 是由于α-或β-珠蛋白基因突变或者缺失使α-或β-珠蛋白链生成缺陷,
最终导致的遗传性溶血性疾病. 同种异体造血干细胞移植是一种能够治愈输血依赖型地贫(TDT) …

Gene editing is a growing gene engineering technique that allows accurate editing of a
broad spectrum of gene‐regulated diseases to achieve curative treatment and also has the …

Genetic inheritance refers to the process by which traits and characteristics are passed from
one generation to the next through the transmission of genetic information. This information …