研究团队首先将人AADC基因装载导到腺相关病毒(AAV)载体中,然后在核磁共振成像技术的帮助下,将AAV病毒精确注射到患者中脑的黑质致密区(SNc)和腹侧被盖区(VTA)。 此次共7名儿童(4-9岁,4女3男)接受这种基因治疗,治疗后2年的随访发现...
截止到2020年11月,Clinical Trials. Gov上登记数据显示,开展AADC缺乏症临床试验的项目为4项,目前正处于I期或者II期临床试验阶段,这些研究均使用AAV2作为体内基因导入平台。除此之外,2019年自治医科大学Kojima et al.发表了一项I期和II期试验结果3,该...
329Radiographic Safety of MRI-Guided AAV2-AADC Infusion in Children With AADC Deficiencydoi:10.1227/neu.0000000000003360_329Aromatic amino-decarboxylase (AADC) deficiency is a rare inherited enzymatic disorder inhibiting dopamine and serotonin production, leading to severe developmental delay, hypotonia, ...
An adeno-associated virus (AAV) vector is used for treating aromatic L-amino acid decarboxylase (AADC) deficiency. The AAV vector is directly injected into human brain through a stereotactic technique. Activity of AADV is thus recovered for solving the problem of movement disorder....
942. Evaluation of Short (8 Weeks) and Long-Term (3 and 5 Years) Gene Transfer Following Brain Delivery of AAV-AADC Vector in Parkinsonian MonkeysGENETIC transformationmt is a cross-disciplinary biomedical journal devoted to publishing the most exciting advances in pharmacology and therapeutics, as...
Objective: We investigated the safety and efficacy of delivery of a viral vector expressing AADC (AAV2-hAADC) to the midbrain in children with AADC deficiency. Design, Setting and Participants: Seven (7) children, aged 4-9 years underwent convection-enhanced delivery (CED) of AAV2-hAADC to...
Keywords AAV-2Convection enhanced deliveryGene transferHeparan sulfate receptorsHeparinMethods of delivering viral vectors, particularly recombinant adeno-associated virus (rAAV) virions, to the central nervous system (CNS) using convection enhanced delivery (CED) are provided. The rAAV virions include ...
Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial. PLoS One. 2017 Feb 6; 12(2): e0169965. doi: 10.1371/journal. pone.0169965. eCollection 2017....
DISTRIBUTION OF AAV VECTORS ENCODING AADC INTENSIFIED BY CONVECTIONMethods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease.BANKIEWICZ, KRYSCUNNINGHAM, JANET...
Methods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease.BANKIEWICZ, KRYS, GARRETT PARK, MD , USCUNNINGHAM, JANET, ALAMEDA, CA , USEBERLING, L., JAMIE, BERKELEY, CA , US...