所有31名接受治疗的严重SCD患者在随访时间为2.0~32.3个月时，均未出现血管闭塞性危象。SCD患者的平均胎儿血红蛋白水平在接受治疗后第四个月时占总血红蛋白水平的40%，并且得到维持。 CRISPR-Cas9基因编辑系统的一种新变体使得它更容易为治疗应用而对大量细胞 ...

Genome editing with various CRISPR-Cas molecule complexes has progressed rapidly in recent years. Hundreds of labs around the world are now working to put these tools to clinical use and are ...

CRISPR’s promise of curing diseases hit a setback when researchers discovered that AZD7648, a molecule designed to improve precision, also caused catastrophic hidden damage to DNA. Instead of perfect ...

MaxCyte’s Flow Electroporation technology delivers CRISPR/Cas-9 gene-editing tools to a patient’s T cells. The technology uses electrical pulses to create temporary pores in the cell membrane, which ...

Genetic modifications in reproductive cells, such as eggs, sperm, or embryos, are commonly referred to as germline editing and are heritable. 1 CRISPR-Cas 9, which stands for clustered regularly ...

Chromosomal rearrangements, such as chromosome segment inversions, may affect the epigenetic landscape as well as gene expression. Different kinds of chromosome segment inversions have been found in ...