From rough to refined: Applying diamond standards to CAR Treg development [0.03%]
从粗到细——以钻石标准促进CAR-Treg细胞的研发
Francesco W Hamilton,Leonardo M R Ferreira
Francesco W Hamilton
Risks of replication-competent retro/lentivirus from associated vector systems: Is it time for a roadmap toward reduced testing? [0.03%]
复制型逆转录/ lent病毒载体系统的风险：是否到了制定减少试验的路线图的时候了？
Daniel Farley,Samuel Stockdale,Charles Moore-Kelly et al.
Daniel Farley et al.
The development of retroviral and lentiviral vectors (RV/LVs) has culminated in third-generation vector systems with excellent safety profiles. However, there has been no significant (re)assessment of RCR/RCL formation risk within RV/LV sys...
Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain [0.03%]
一种C端延长微型 dystrophin的增强治疗潜力
Steven J Foltz,Randolph Qian,Jason Yang et al.
Steven J Foltz et al.
Gene replacement therapy is becoming a therapeutic option for patients with Duchenne muscular dystrophy. Truncated dystrophins that can be expressed from adeno-associated viral (AAV) vectors have been designed and shown to retain many, thou...
Structuring of lipid nanoparticle mRNA formulations at acidic and neutral pH: X-ray scattering and molecular dynamics studies [0.03%]
脂纳米粒mRNA递送体系的结构研究：酸性和中性pH下脂纳米粒体系的散射和分子动力学模拟研究
Amali G Guruge,Yujia He,Andrew J Clulow et al.
Amali G Guruge et al.
mRNA lipid nanoparticles (LNPs) effectively deliver mRNA into cells. However, the structure of these particles remains poorly understood. LNP formulations are manufactured by initially forming nanoparticles at pH 4, to incorporate the RNA, ...
A novel gene delivery platform based on a commensal human anellovirus demonstrates transduction in multiple tissue types [0.03%]
一种新型的基因递送平台基于共生的人类环状病毒，在多种组织类型中都表现出转导性
Cato Prince,George Bounoutas,Bolu Zhou et al.
Cato Prince et al.
Anelloviridae is a family of non-enveloped viruses with negative-sense, circular, single-stranded DNA genomes that infect vertebrates and are a ubiquitous component of the human virome. The non-pathogenic and commensal nature of anellovirus...
Paul J H Nijhuis,Maurits Romijn,Roy Honing et al.
Paul J H Nijhuis et al.
Multiple sclerosis (MS) is a neurological disease characterized by demyelinating lesions in the CNS. This study investigated whether a minimally invasive adeno-associated virus (AAV) vector (AAV.PHP.eB) can direct transgene expression in CN...
Characterization of adeno-associated viral transduction of retinal ganglion cells in adult and old mice [0.03%]
表征腺相关病毒转导成年和老年小鼠视网膜神经节细胞的特点
Delaney Cm Henderson,Balwantray C Chauhan
Delaney Cm Henderson
Adeno-associated viral (AAV) vectors are being used increasingly in developing treatments for age-related ocular diseases, including those impacting retinal ganglion cells (RGCs), the output neurons of the eye. However, the effects of persi...
Genome-replicating HC-AdV: A novel high-capacity adenoviral vector class featuring enhanced in situ payload expression [0.03%]
具有增强原位负载表达的新颖型高容量腺病毒载体基因组复制HC-AdV
Jonas Kolibius,Fabian Weiss,Patrick C Freitag et al.
Jonas Kolibius et al.
High-capacity adenoviral (HC-AdV) vectors offer large transgene capacities and long-term expression of therapeutics but require high doses due to limited transgene expression. In contrast, replication-competent AdV (RC-AdV) vectors enhance ...
Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates [0.03%]
经脑池内递送后恒河猴体内AAV1、AAV5、AAV9和AAVDJ血清型的分布odynamics of neurofilament light chain in cerebrospinal fluid and plasma over two decades in healthy individuals
Takuro Okai,Sho Sato,Hironobu Yasuno et al.
Takuro Okai et al.
Delivering drugs effectively to the central nervous system (CNS) is a major challenge in drug development, including adeno-associated virus (AAV) gene therapy. The cerebrospinal fluid (CSF) circulates through the ventricular system and the ...
Elevated tripeptidyl-peptidase 1 corrects multiple disease phenotypes in a mouse model of juvenile neuronal ceroid lipofuscinosis [0.03%]
三肽基肽酶1的上调可纠正一例儿童神经元 ceroid 淀粉样变病小鼠模型中的多种疾病表型
Whitney Banach-Petrosky,Katherine E Larrimore,Elizabeth H Sleat et al.
Whitney Banach-Petrosky et al.
Juvenile neuronal ceroid lipofuscinosis (JNCL) is a neurodegenerative lysosomal storage disease caused by the loss of the endolysosomal transmembrane protein, CLN3. The main protein component of lysosomal storage material in JNCL is subunit...