Ultrasound-Targeted Microbubble Destruction Delivery of Insulin-Like Growth Factor 1 cDNA and Transforming Growth Factor Beta Short Hairpin RNA Enhances Tendon Regeneration and Inhibits Scar Formation In Vivo [0.03%]
靶向微泡破坏递送胰岛素样生长因子1和转化生长因子Beta短发夹RNA干扰核酸促进肌腱再生和抑制瘢痕形成
Xi Xiang,Qianying Leng,Yuanjiao Tang et al.
Xi Xiang et al.
Ultrasound-targeted microbubble destruction (UTMD), which has been successfully used for the treatment of many diseases, offers a promising noninvasive approach for target-specific gene delivery. This study investigated the UTMD delivery of...
Varinostat Alters Gene Expression Profiles in Aortic Tissues from ApoE-/- Mice [0.03%]
富马酸瓦利诺司特改变ApoE基因敲除小鼠主动脉组织的基因表达谱
Yicong Ye,Xiliang Zhao,Yiyun Lu et al.
Yicong Ye et al.
Atherosclerosis (AS) is a complex, chronic inflammatory disease that is characterized by plaque buildup within arterial vessel walls. Preclinical trials have suggested that vorinostat, a pan-histone deacetylase inhibitor (HDACi), reduces va...
Toxicology and Pharmacology of an AAV Vector Expressing Codon-Optimized RPGR in RPGR-Deficient Rd9 Mice [0.03%]
用于治疗RPGR缺陷型Rd9小鼠的表达优化密码子的RPGR的AAV载体的安全性及药代动力学研究
Chunjuan Song,Thomas J Conlon,Wen-Tao Deng et al.
Chunjuan Song et al.
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis p...
James M Wilson
James M Wilson
Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia Investigated in an Exploratory Dose-Escalation Trial [0.03%]
视锥环核通道亚单位A3基因相关全色盲患者玻璃体内注射rAAV.hCNGA3的探索性剂量递增试验：方法和方案研究-安全性和有效性（单次给药）
Nadine A Kahle,Tobias Peters,Ditta Zobor et al.
Nadine A Kahle et al.
Achromatopsia is an autosomal recessively inherited congenital defect characterized by a lack of cone photoreceptor function, leading to severely impaired vision. In this clinical study, achromatopsia patients were treated with a single sub...
MiRNA-451 Inhibits Glioma Cell Proliferation and Invasion Through the mTOR/HIF-1α/VEGF Signaling Pathway by Targeting CAB39 [0.03%]
微小核糖核酸-451通过靶向CAB39抑制胶质瘤细胞增殖和侵袭 механизма(signalling pathway)换成信号通路
Yang Nan,Hongbao Guo,Liyun Guo et al.
Yang Nan et al.
MicroRNAs (miRNAs) are widely expressed and regulate most biological functions. According to several research groups, miR-451 expression is decreased in glioma cells. A previous study also confirmed that miRNA-451 inhibits the PI3K/AKT sign...
Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease [0.03%]
帕金森病 lentiviral 基因疗法 ProSavin 长期随访研究
Stéphane Palfi,Jean Marc Gurruchaga,Hélène Lepetit et al.
Stéphane Palfi et al.
Parkinson's disease is typically treated with oral dopamine replacement therapies. However, long-term use is complicated by motor fluctuations from intermittent stimulation of dopamine receptors and off-target effects. ProSavin, a lentivira...
Gene Therapy for Osteoarthritis: Pharmacokinetics of Intra-Articular Self-Complementary Adeno-Associated Virus Interleukin-1 Receptor Antagonist Delivery in an Equine Model [0.03%]
马模型中玻璃体内注射自互补腺相关病毒白细胞介素-1受体拮抗剂的药代动力学研究：关节内基因治疗骨关节炎的研究
Rachael S Watson Levings,Ted A Broome,Andrew D Smith et al.
Rachael S Watson Levings et al.
Toward the treatment of osteoarthritis (OA), the authors have been investigating self-complementary adeno-associated virus (scAAV) for intra-articular delivery of therapeutic gene products. As OA frequently affects weight-bearing joints, ph...