碱基编辑疗法对抗T细胞白血病效果显著

Core Insights - A new gene therapy named BE-CAR7 has been developed by a joint team from University College London and Great Ormond Street Hospital, showing significant effectiveness against T-cell acute lymphoblastic leukemia (T-ALL) [1][2] - The therapy utilizes base editing technology, a more advanced version of CRISPR, allowing precise single-letter modifications in DNA without cutting the DNA strands, thus reducing the risk of chromosomal damage [1][2] Group 1: Treatment Process and Results - BE-CAR7 cells are engineered from healthy donor T-cells, modified to recognize and target CD7 markers on leukemia T-cells [1] - After injection, these modified cells proliferate and aim to destroy all T-cells, including leukemia cells, with a successful clearance of leukemia lesions leading to potential bone marrow transplants for immune system reconstruction [2] - Among treated patients, 82% achieved deep remission, allowing for stem cell transplants, and 64% maintained a disease-free status, with some patients remaining disease-free for three years [2] Group 2: Patient Case and Broader Implications - The first beneficiary of this therapy is a 16-year-old girl from Leicester, who, after previous treatments failed, became the first global patient to receive base editing therapy and successfully cleared her cancer cells within a month [2] - This breakthrough offers hope for the approximately 20% of T-cell leukemia patients who do not respond to standard treatments, illuminating a promising path in the field of cancer therapy [3]