Durable tissue-specific transgene expression in newborn mice following intraperitoneal delivery of non-cytotoxic HSV vectors [0.03%]
非细胞毒性HSV载体在新生小鼠腹腔给药后实现持久的组织特异性转基因表达
Yoshitaka Miyagawa,Motoyo Maruyama,Atsushi Sakai et al.
Yoshitaka Miyagawa et al.
This report describes the distribution and transgene expression of two non-cytotoxic, replication-defective (rd) herpes simplex virus (HSV) vectors, JΔNI7 and JΔNI8, following intraperitoneal delivery to newborn mice. The two vectors are ...
Simple, specific, rapid, and pharmacopoeia-compliant qPCR approach for the detection of mycoplasma in biopharmaceuticals [0.03%]
一种简单、特异、快速且符合药典的生物制药支原体qPCR检测方法
Sergio Dos Santos,Emilie Lespinasse,Baptiste Bonnet et al.
Sergio Dos Santos et al.
Cell substrate utilized in the production of biologics intended for human use need to be cleared from mycoplasma contaminant as described in pharmacopoeia. While the gold-standard method for mycoplasma detection involves lengthy microbiolog...
Harnessing mRNA-lipid nanoparticles as innovative therapies for autoimmune diseases [0.03%]
利用mRNA-脂质纳米颗粒作为自身免疫性疾病创新疗法
Rose Razavi,Michael Kegel,Jenna Muscat-Rivera et al.
Rose Razavi et al.
Autoimmune diseases are chronic conditions where the immune system mistakenly attacks healthy tissues, leading to potentially debilitating symptoms that require lifelong management. There are no cures for autoimmune diseases, and new treatm...
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study [0.03%]
HB-201慢性B型血友病患者导入期AAV5预先存在抗体的自然史：HOPE-B III期临床试验结果
Robert Klamroth,Michael Recht,Nigel S Key et al.
Robert Klamroth et al.
Testing for binding or neutralizing antibodies (NAbs) to adeno-associated virus (AAV) is part of the laboratory assessment of people with hemophilia considering AAV-based gene therapy. We evaluated the natural history of NAb titers to AAV s...
Nanoparticle delivery of AMPK activator 991 prevents its toxicity and improves muscle homeostasis in Duchenne muscular dystrophy [0.03%]
纳米颗粒递送AMPK激活剂991预防其毒性并改善杜氏肌营养不良的肌肉稳态
Ilaria Andreana,Ananga Ghosh,Mathieu Repellin et al.
Ilaria Andreana et al.
Muscular dystrophies, such as Duchenne muscular dystrophy (DMD), are caused by permanent muscle injuries leading to chronic inflammation, with macrophages harboring an altered inflammatory profile contributing to fibrosis through the secret...
Effects of CRISPR-Cas9-mediated FOXP3 knockout on CAR T cell potency [0.03%]
基于CRISPR-Cas9的FOXP3基因敲除对CAR-T细胞疗效的影响
Lena Peter,Martí Farrera-Sal,Ferhat Ali Yaman et al.
Lena Peter et al.
Persistent antigen stimulation and inflammatory environments drive exhaustion, senescence, and activation-induced cell death, impairing both endogenous and therapeutic T cells. Understanding the mechanisms underlying T cell dysfunction is c...
To be or not to PHP.eB? Potential strategies for therapeutically "attacking" multiple sclerosis [0.03%]
是还是否？针对多发性硬化症的潜在治疗策略
Jessica A Herstine,Benjamin L L Clayton
Jessica A Herstine
HLA-A2 CAR/IL-2-CISC engineered Treg display robust in vitro and in vivo antigen-specific regulatory function [0.03%]
同种异体抗原递呈型CAR-Treg细胞表现出良好的异体相容性及体内体外特异性免疫抑制功能
Subhash K Tripathi,Annaiz Grimm,Noelle P Dahl et al.
Subhash K Tripathi et al.
Chimeric antigen (Ag) receptor-expressing T regulatory cells (CAR-Treg) offer therapeutic potential for treating autoimmunity, allograft rejection, and graft-versus-host disease (GvHD). HLA-A∗02 (A2) CAR (A2CAR)-expressing natural Treg hav...
Perineural delivery of AAV2/9 in non-human primates is a safe and efficient route for gene therapy in Charcot-Marie-Tooth diseases [0.03%]
非人灵长类的外周神经AAV2/9载体递送安全有效，适用于遗传性感觉运动周围神经病基因治疗临床转化研究
Julie Espallergues,Julien Cadiet,Fouzia Souab et al.
Julie Espallergues et al.
AAV-based gene therapy represents an attractive treatment for hereditary peripheral neuropathies Charcot-Marie-Tooth diseases. We recently showed that AAV2/9 vector-expressing GFP, locally injected into mouse and rat sciatic nerves, transdu...
AAV-mediated peripheral scFv's administration to reduce cerebral tau in adult P301S transgenic mice: Mono-vs. combination therapy [0.03%]
利用AAV介导的周围scFv给药减少成人P301S转基因小鼠脑tau蛋白：单次与联合疗法
Sebica Katel,Julia Cicalo,Valeria Vasciaveo et al.
Sebica Katel et al.
Tau is a primary target for immunotherapy in Alzheimer's disease (AD). Recent studies have shown the potential of anti-tau fragment antibodies in lowering pathological tau levels in vitro and in vivo. Here, we compared the effects of single...