Self-Complementary Adeno-Associated Virus-Mediated Interleukin-1 Receptor Antagonist Gene Delivery for the Treatment of Osteoarthritis: Test of Efficacy in an Equine Model [0.03%]
自我互补腺相关病毒介导的白细胞介素-1受体拮抗基因治疗骨关节炎：马模型的有效性试验
Rachael S Watson Levings,Andrew D Smith,Ted A Broome et al.
Rachael S Watson Levings et al.
The authors are investigating self-complementary adeno-associated virus (scAAV) as a vector for intra-articular gene-delivery of interleukin-1 receptor antagonist (IL-1Ra), and its therapeutic capacity in the treatment of osteoarthritis (OA...
Zuzana Kočí,Tomáš Boráň,Petr Krůpa et al.
Zuzana Kočí et al.
Advanced therapy medicinal products (ATMPs) represent a new generation of biopharmaceuticals that comprise gene therapy medicinal products (GTMPs), somatic cell therapy products (CTMPs), tissue engineered products (TEPs), and combined advan...
Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease [0.03%]
治疗X连锁慢性肉芽肿病的慢病毒载体G1XCGD的非临床有效性及安全性研究
Christian Brendel,Michael Rothe,Giorgia Santilli et al.
Christian Brendel et al.
Chronic granulomatous disease (CGD) is a debilitating primary immunodeficiency affecting phagocyte function due to the absence of nicotinamide dinucleotide phosphate (NADPH) oxidase activity. The vast majority of CGD patients in the Western...
A Multicenter, Double-Blind, Phase III Clinical Trial to Evaluate the Efficacy and Safety of a Cell and Gene Therapy in Knee Osteoarthritis Patients [0.03%]
一项关于细胞及基因疗法治疗膝关节骨关节炎有效性和安全性的多中心双盲III期临床试验
Myung-Ku Kim,Chul-Won Ha,Yong In et al.
Myung-Ku Kim et al.
The aim of this study was to test the clinical efficacy of TissueGene-C (TG-C), a cell and gene therapeutic for osteoarthritis consisting of non-transformed and transduced chondrocytes (3:1) retrovirally transduced to overexpress transformi...
A Multicenter, Double-Blind, Phase III Clinical Trial to Evaluate the Efficacy and Safety of a Cell and Gene Therapy in Knee Osteoarthritis Patients [0.03%]
一种细胞及基因治疗方法治疗膝骨关节炎的多中心随机双盲III期临床试验疗效和安全性评价研究
Myung-Ku Kim,Chul-Won Ha,Yong In et al.
Myung-Ku Kim et al.
The aim of this study was to test the clinical efficacy of TissueGene-C (TG-C), a cell and gene therapeutic for osteoarthritis consisting of non-transformed and transduced chondrocytes (3:1) retrovirally transduced to overexpress transformi...
Gene Therapy Briefs [0.03%]
基因治疗简报
Alex Philippidis
Alex Philippidis
James M Wilson
James M Wilson
Practical Implications of Factor IX Gene Transfer for Individuals with Hemophilia B: A Clinical Perspective [0.03%]
从临床角度分析凝血因子IX基因治疗血友病B的实践意义
Wolfgang Miesbach,Eileen K Sawyer
Wolfgang Miesbach
Gene therapy for severe hemophilia is on the cusp of entering clinical practice. However, there is limited clinical experience in this area given that gene transfer is a relatively recent technology. Therefore, this clinical perspective art...
Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans [0.03%]
人类中针对两种F血清型AAV的中和抗体的血清阳性率低
Jeff L Ellsworth,Michael O&#x;Callaghan,Hillard Rubin et al.
Jeff L Ellsworth et al.
To assess the therapeutic utility of AAVHSC15 and AAVHSC17, two recently described Clade F adeno-associated viruses (AAVs), the seroprevalence of neutralizing antibodies (NAbs) to these AAVs was assessed in a representative human population...
Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans [0.03%]
人源中靶向AAV F亚群的中和抗体低阳性率
Jeff L Ellsworth,Michael O&#x;Callaghan,Hillard Rubin et al.
Jeff L Ellsworth et al.
To assess the therapeutic utility of AAVHSC15 and AAVHSC17, two recently described Clade F adeno-associated viruses (AAVs), the seroprevalence of neutralizing antibodies (NAbs) to these AAVs was assessed in a representative human population...