A rescue fanconi anemia humanized mouse model with endogenous FA mutation and high human hematopoietic stem cell chimerism [0.03%]
一种带有本源性Fanconi贫血症突变和高比例人类造血干细胞嵌合体的人类免疫缺陷小鼠模型
Seunga Choo,Alvin H Tong,Emily Fields et al.
Seunga Choo et al.
Autologous transplantation of ex vivo gene-modified/corrected hematopoietic stem cells (HSCs) offers a definitive therapeutic approach to restore hematopoiesis in Fanconi anemia (FA) patients. However, this approach not only requires ex viv...
Early postnatal expression mitigates immune responses to Cas9 in the murine central nervous system [0.03%]
早发Cas9表达减轻小鼠中枢神经系统中的免疫反应
Robert Duba-Kiss,David R Hampson
Robert Duba-Kiss
A barrier in the development of adeno-associated virus (AAV) gene therapy is the immunogenicity of the AAV particles, and in some cases, the expressed transgene. The immunogenic risk is heightened when exogenous proteins, such as prokaryoti...
Are viral vector-mediated therapies compatible with aberrant glycosylation? [0.03%]
病毒载体介导的治疗与异常糖基化相容吗？
I J J Muffels,R Budhraja,S Radenkovic et al.
I J J Muffels et al.
The ability of adeno-associated viruses (AAVs) to transduce host cells relies on interactions with glycan moieties on the cellular surface. Consequently, disrupted protein glycosylation, which is seen in a range of neurodevelopmental and ne...
Development of VNX-101, an adeno-associated virus with less immunogenicity and efficient long-term expression of a CD19 T cell engager [0.03%]
VNX-101载体的开发：一种免疫原性较低且能有效长期表达CD19T细胞衔接器的腺相关病毒
Mark A Currier,Allen Reha,Brian Hutzen et al.
Mark A Currier et al.
We previously described the use of recombinant adeno-associated virus (AAV) gene therapy to achieve off-the-shelf, long-term in vivo T cell engagement for CD19+ B cell malignancies following a single dose by expressing a transgene encoding ...
AAV-shDUX4 provides short-term benefits but limited long-term efficacy in a DUX4 mouse model of FSHD [0.03%]
AAV-shDUX4治疗短期内可改善FSHD小鼠模型症状但长期疗效有限
S Sohn,S Reid,M Bowen et al.
S Sohn et al.
The aberrant expression of the toxic transcription factor DUX4 in skeletal muscle is a hallmark of facioscapulohumeral muscular dystrophy. Effective therapeutic strategies will likely require the inhibition of DUX4, with adeno-associated vi...
Rapid cochlear gene therapy in adult deaf mice: Vglut3 rescue via AAV8 achieves day-1 hearing restoration [0.03%]
通过AAV8实现快速耳蜗基因治疗：Vglut3的拯救在聋哑小鼠上实现第1天恢复听力
Ting Zhang,Rongqun Zhai,Mengli Liu et al.
Ting Zhang et al.
Genetic hearing loss, caused by mutations in critical auditory genes, has seen promising advances through gene therapy, yet the temporal dynamics of early-stage auditory functional recovery and therapeutic transgene expression patterns foll...
EF1α, rather than CMV promoter, is suitable for luciferase tag expression in target cells for in vitro cytotoxicity assays of CAR-T cells [0.03%]
CAR-T细胞体外毒性试验中目标细胞适用萤火虫荧光素酶标签表达的启动子考察
Rui Hou,Zejun Zhang,Huan Li et al.
Rui Hou et al.
Precise assessment of the cytotoxic activity of engineered immune cell therapeutics, such as chimeric antigen receptor-engineered T (CAR-T) cells, is essential for their development and quality control. However, luciferase (Luc)-based viabi...
Development of cell-based assay for detecting replication-competent adeno-associated virus by qPCR [0.03%]
基于细胞的定量PCR检测重组腺相关病毒的复制能力的方法学建立
Aishwarya Shevade,John S Reeves,Hanne Bak et al.
Aishwarya Shevade et al.
Recombinant, non-replicative adeno-associated virus (AAV) containing a therapeutic gene of interest (GOI) is widely used as a vector for gene therapy. One way to manufacture AAV is through triple-transfection of HEK293 cells, with plasmids ...
George Pamenter,Danyal H Rahim,Ciaran Lamont et al.
George Pamenter et al.
The growing demand for lentiviral vectors (LVs) in cell and gene therapies has highlighted significant challenges in large-scale LV production, particularly low product recovery. Anion-exchange chromatography (AIEX) is widely used for LV ca...
Considerations in biodistribution evaluation of iPSC-derived cell therapy: A pancreatic islet cell case study [0.03%]
诱导多能干细胞治疗的生物分布评估考虑因素：胰岛细胞案例研究
Miyu Nakayama,Yuu Moriya,Hikaru Ueno et al.
Miyu Nakayama et al.
Induced pluripotent stem cells (iPSCs) have substantial transformative potential in regenerative medicine, enabling tissue repair and restoration. However, their clinical application is limited by tumorigenic risks owing to their pluripoten...