Jul 27, 2021 · 综合实验结果，课题组证实AAV介导的CAR分子基因治疗（AAV-mediated in vivo CAR gene therapy，ACG）能够在体内发挥特异性杀伤功能，从而达到类似传统CAR-T细胞治疗的效果。

aav由包含单链dna基因组的非包膜蛋白衣壳组成，因此它的遗传修饰通常是短暂的，特别是在增殖细胞中（如活化的淋巴细胞）。 在与它们的靶受体相互作用后，网格蛋白介导内吞作用和胞内运输（这些过程的细节由衣壳的血清型决定），AAV进入细胞核并释放单链 ...

该课题组利用AAV（ 腺相关病毒 ）载体同时将多个Cpf1/Cas12a Crisper RNA和修复模板传入T细胞，高效构建了一个稳定的CAR-T细胞，该细胞(KIKO CAR-T细胞)具有同源修复敲入和免疫检查点敲除的双重功能。

Jun 1, 2022 · In this study, we optimized the protocol for rAAV6 transduction of primary T cells, significantly improved the expression efficiency of the rAAV6 delivered CAR gene, and successfully generated rAAV6-based CAR-T cells (AAV-CAR-T).

Jun 23, 2021 · Here, we describe a novel approach in which human CAR T cells can be generated within the host upon injecting an Adeno-associated virus (AAV) vector carrying the CAR gene, which we call AAV...

Jan 16, 2023 · 近年来，基于基因工程改造的CAR-T细胞疗法在治疗血液类癌方面展现了强大疗效，FDA先后批准了6款CAR-T疗法上市，研究人员也在积极探索CAR-T治疗实体瘤的潜力。 在构建CAR-T细胞的过程中，通常使用 逆转录病毒 或 慢病毒 载体，但是这两类病毒载体会随机整合到细胞基因组中，这种随机整合可能导致T细胞功能异质性、转入基因沉默，甚至可能激活细胞癌变，这限制了细胞治疗产品的有效性和安全性。 近年来，随着CRISPR基因编辑技术的进步，我 …

Feb 25, 2019 · By leveraging trans-activating CRISPR (clustered regularly interspaced short palindromic repeats) RNA (tracrRNA)-independent CRISPR–Cpf1 systems with adeno-associated virus (AAV), we were able to...

Aug 31, 2022 · Here we successfully developed a two-in-one approach to generate non-viral, gene-specific targeted CAR-T cells through CRISPR–Cas9. Using the optimized protocol, we demonstrated feasibility in a...

Feb 15, 2021 · As envisioned, the AAV vector generated enough CAR T cells in vivo and thus 99 caused effective tumor regression and produced antitumor immunological characteristics. In

In the associated publication, we describe a new method for generating CAR-T cells using a combination of mRNA electroporation for LbCpf1 and AAV6 for crRNA and homology-directed repair (HDR) template. We achieved simple yet highly efficient targeting of both HDR-mediated CAR-T knockin and immune checkpoint gene knockout (KIKO).