Characterization and quantitation of baculoviral DNA in rAAV vectors produced in Sf9 cells [0.03%]
Sf9细胞中产生的rAAV载体中的杆状病毒DNA的表征和定量
Xushan Wang,Andrew Pla,Vedud Purde et al.
Xushan Wang et al.
Recombinant adeno-associated virus (rAAV) vectors are widely used in gene therapy due to their ability to transduce various cell types and tissues, sustained gene expression, and relatively safe profile. However, the production of rAAV vect...
In vivo delivery of a fluorogenic antisense oligonucleotide into human leukocytes in a humanized mouse model system [0.03%]
人源化小鼠模型系统中的人白细胞体内递送荧光嵌色反义寡核苷酸
Nathan B Seidel,Saurav S Rout,Beverly Z Packard et al.
Nathan B Seidel et al.
Development of oligonucleotide-based therapeutics has been limited by the lack of effective in vivo delivery vehicles. We previously showed that the presence of an H-type excitonic dimer formed by the covalent binding of two fluorophores wi...
Development of high-throughput analytical methods for characterization and quantitation of rAAV genome integrity [0.03%]
高通量分析方法的发展用于表征和定量rAAV基因组完整性
Xushan Wang,Zhaohui Yang,Andrew Pla et al.
Xushan Wang et al.
Recombinant AAV (rAAV) vectors are a leading viral vector for gene therapy. Viral genome (Vg) titer is the primary method to determine potency of rAAV and dosing in preclinical/clinical studies. However, the rAAV genome comprises a heteroge...
Morphological changes induced by buffer exchange during preparation of mRNA-lipid nanoparticles occur in a stepwise manner as pH is elevated [0.03%]
缓冲液交换过程中诱导的mRNA-脂质纳米颗粒形态变化随着pH值升高而逐步进行
Yujia He,Emily H Pilkington,Hee Jung Kang et al.
Yujia He et al.
Initial entrapment of nucleic acids in lipid nanoparticles (LNPs) is dependent on the use of ionizable cationic lipids, which draw nucleic acids into lipid particles at low pH in the presence of ethanol. Manufacturing of fully formed LNPs i...
Targeted inhibition of ELANE expression using adenine base editing to treat severe congenital neutropenia [0.03%]
利用腺嘌呤碱基编辑特异性抑制ELANE表达以治疗严重先天性中性粒细胞减少症
Betül Findik,Benjamin Dannenmann,Franka Bernhard et al.
Betül Findik et al.
Autosomal dominant mutations in ELANE (elastase, neutrophil expressed) cause severe congenital neutropenia (CN) and cyclic neutropenia (CyN). Inhibiting ELANE expression, either by CRISPR-Cas9-mediated ELANE knockout or promoter targeting u...
AAV-PHP.eB achieves superior neuronal transduction over AAV9 in pigtail macaques following intracerebroventricular administration [0.03%]
经脑室内给药后，AAV-PHP.eB载体在断尾猴大脑中实现优于AAV9的神经元转导效应
Michal G Fortuna,Lyle H Nyberg,Naz Taskin et al.
Michal G Fortuna et al.
Adeno-associated virus (AAV) vectors are pivotal in gene therapy for neurological disorders due to their ability to enable long-term gene expression in the central nervous system (CNS). However, transducing larger brains, such as those of n...
Nanobody-based gene therapy targeting complement component C3 reduces choroidal neovascularization in mice [0.03%]
针对补体成分C3的纳米抗体基因治疗可减少小鼠脉络膜新生血管生成
Emilie Grarup Jensen,Thomas Stax Jakobsen,Gloriane Schnabolk et al.
Emilie Grarup Jensen et al.
Wet age-related macular degeneration (wAMD) is a leading cause of vision loss and is characterized by choroidal neovascularization (CNV). Current CNV management requires multiple treatments and lacks long-term efficiency, creating a need fo...
Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following AAV gene therapy [0.03%]
免疫抑制可减少经腺相关病毒基因治疗后食蟹猴背根神经节和外周神经的病理变化
Branka Grubor,Kate L Henry,Su Jing Chan et al.
Branka Grubor et al.
Administration of adeno-associated virus (AAV) gene therapies via blood or cerebrospinal fluid (CSF) in non-human primates (NHPs) can lead to degeneration of dorsal root ganglion (DRG) neurons and nerve fibers in the spinal cord and periphe...
When pull ulanase needs a little push: MyoAAV capsids enhance gene therapy for GSD IIIa [0.03%]
当拉取过程需要一点推动时：MyoAAV衣壳增强GSD IIIa基因治疗
Sree Venigalla,Christina A Pacak
Sree Venigalla
HBVZ10, an AAV8 vector-based new HBV therapy candidate for cccDNA elimination [0.03%]
一种新型乙肝治疗候选药物HBVZ10可消除共价闭合环状DNA（cCCCDNA）
Bai-Hua Zhang,Yuanping Zhou,Stephen Horrigan et al.
Bai-Hua Zhang et al.
Eliminating hepatitis B virus (HBV) covalently closed circular DNA (cccDNA) remains a major challenge, requiring innovative treatment strategies and drug candidates. Clinical studies reveal that wild-type HBV in the blood is often replaced ...