GroβT rapidly and reliably mobilizes primitive hematopoietic stem and progenitor cells in nonhuman primates [0.03%]
GroβT快速而可靠地动员非人灵长类动物的原始造血干细胞和祖细胞
Andrea Repele,Dnyanada Pande,Mark R Enstrom et al.
Andrea Repele et al.
Autologous hematopoietic stem cell (HSC) gene therapy has gone through remarkable advancements in recent years, especially for the treatment of sickle cell disease (SCD). However, the collection of HSCs from SCD patients requires unique con...
High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa [0.03%]
高活性MyoAAV衣壳体通过小鼠GSD IIIa模型增强骨骼肌校正
Kuo-An Liao,Sang-Oh Han,Mercedes Barzi et al.
Kuo-An Liao et al.
Glycogen storage disease type IIIa (GSD IIIa) affects multiple tissues, including liver, heart, and skeletal muscles. We recently reported that an adeno-associated virus serotype 9 vector expressing pullulanase, a bacterial glycogen debranc...
AAV hamartin gene therapy in a stochastic, cerebral mouse model of tuberous sclerosis type 1 [0.03%]
用于随机突变型I型结节性硬化的小鼠模型的腺相关病毒hamartin基因治疗
Edwina Abou Haidar,Shilpa Prabhakar,Alexandra L Geffrey et al.
Edwina Abou Haidar et al.
Tuberous sclerosis complex (TSC) is a dominantly inherited disease in which most individuals are born with one defective allele encoding for either hamartin (TSC1) or tuberin (TSC2), with a somatic loss of the other allele leading to abnorm...
iPSC-hepatocyte organoids as a novel platform to predict AAV gene therapy efficacy [0.03%]
iPSC-肝细胞类器官作为预测AAV基因治疗效果的新平台
Estelle Berreur,Giacomo Lazzaroni,Cyrill Roth et al.
Estelle Berreur et al.
Adeno-associated virus (AAV) vectors are widely used in gene therapy, particularly for liver-targeted treatments. However, predicting human-specific outcomes, such as transduction efficiency and hepatotoxicity, remains challenging. Reliable...
A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease [0.03%]
结合骨骼肌顺式作用元件的肌动性AAV载体改善Pompe病小鼠模型糖原清除率
P Sellier,F Collaud,Y Krimi Benchekroun et al.
P Sellier et al.
Pompe disease is a glycogen storage disorder caused by mutations in the acid α-glucosidase (GAA) gene, leading to reduced GAA activity and glycogen accumulation in heart and skeletal muscles. Enzyme replacement therapy with recombinant GAA...
Advancing adeno-associated virus for Duchenne muscular dystrophy treatment: Moving beyond rodent models [0.03%]
迈向杜氏肌营养不良症治疗的腺相关病毒载体研究：超越啮齿类动物模型
Oluwatayo F Ikotun,Elizabeth M McNally,Melissa J Spencer
Oluwatayo F Ikotun
Species-specific gene expression manipulation in humanized livers of chimeric mice via siRNA-encapsulated lipid nanoparticle treatment [0.03%]
通过siRNA包封的脂质纳米颗粒治疗在嵌合小鼠的人工肝脏中进行物种特异性基因表达操纵
Kazuto Yamazaki,Kenji Kubara,Go Sugahara et al.
Kazuto Yamazaki et al.
Liver-humanized chimeric mice (PXB-mice) are widely utilized for predicting human pharmacokinetics (PK) and as human disease models. However, residual metabolic activity of mouse hepatocytes in chimeric mice can interfere with accurate huma...
Sijia Xu,Zhenzhen Hu,Fenglin Song et al.
Sijia Xu et al.
Lipid nanoparticles (LNPs) are lead non-viral vectors for delivering nucleic acids. LNPs can efficiently encapsulate nucleic acids, protect them from degradation, enhance cellular uptake and induce endosome escape, which show high transfect...
Preclinical studies of an AAV8-CYP4V2 gene therapy VGR-R01 for the treatment of Bietti crystalline dystrophy [0.03%]
用于治疗贝特氏结晶体病的AAV8-CYP4V2基因疗法VGR-R01的药物临床前研究
Wenjing Luo,Lu Guo,Liang Lu et al.
Wenjing Luo et al.
Bietti crystalline dystrophy (BCD) is an autosomal recessive disorder caused by loss-of-function mutations in the CYP4V2 gene, characterized by crystal-like lipid deposits in the retina, progressive photoreceptor loss, and retinal pigment e...
AAV2 delivery of the saCas9 gene results in presentation of an HLA-A∗02:01-restricted T cell epitope potent to induce T cell cytotoxicity [0.03%]
腺相关病毒(AAV)介导的saCas9基因递送可导致产生呈现在HLA-A*02：01限制性T细胞表位并能够诱导细胞毒性的T细胞
Susana S Najera,Annalisa Nicastri,Sojin Bing et al.
Susana S Najera et al.
In vivo genome editing with CRISPR-Cas9 systems is generating worldwide attention and enthusiasm for the possible treatment of genetic disorders. However, the consequences of potential immunogenicity of the bacterial Cas9 protein and the AA...