Lipid nanoparticle-based non-viral in situ gene editing of congenital ichthyosis-causing mutations in human skin models [0.03%]
基于脂质纳米颗粒的非病毒原位基因编辑人类皮肤模型中的先天性鱼鳞病致病突变
Dilem Ceren Apaydin,Gaurav Sadhnani,Tiffany Carlaw et al.
Dilem Ceren Apaydin et al.
Autosomal recessive congenital ichthyosis (ARCI) refers to a group of rare, highly debilitating skin disorders that significantly impair patients' quality of life and lack any effective treatment options. Here, we report clinically relevant...
High-content screening of organoids reveals the mechanisms of human pancreas acinar specification [0.03%]
高内涵筛选揭示人胰腺腺泡细胞分化的分子机制
Rashmiparvathi Keshara,Karolina Kuodyte,Antje Janosch et al.
Rashmiparvathi Keshara et al.
Organoids derived from pluripotent stem cells have emerged as powerful models to study human development. To investigate signaling pathways regulating human pancreas differentiation and morphogenesis, we developed a high-content, image-base...
Challenges and opportunities for human Organ Chips in FDA assessments and pharma pipelines [0.03%]
人体器官芯片在FDA评估和制药管线中的挑战与机遇
Donald E Ingber
Donald E Ingber
The Food and Drug Administration recently announced a plan to phase out animal-testing requirements for drug assessments based on advances in new approach methodologies, including human organ-on-a-chip (Organ Chip) microfluidic culture tech...
Activation of a branched-chain amino acid rheostat restores replication-dependent hematopoietic stem cell fitness [0.03%]
激活支链氨基酸通路恢复造血干细胞的复制依赖型健壮性
James Bartram,Sydney Treichel,Baobao Annie Song et al.
James Bartram et al.
Adult hematopoietic stem cells (HSCs) sustain the lifelong production of all mature blood and immune cells. HSCs possess extensive regenerative potential, but their self-renewal is limited. A long-standing question has been why replicative ...
Nanoengineered extrusion-aligned tract bioprinting enables functional repair of spinal cord injuries [0.03%]
纳米工程挤出对准生物打印实现脊髓损伤的功能修复
Wenhui Huang,Shuxian Chen,Kai Li et al.
Wenhui Huang et al.
Spinal cord repair demands biomaterials that replicate the aligned axonal architecture and mechanical softness of native tissue. However, most current scaffolds fail to support three-dimensional alignment and neuronal differentiation of hum...
Development of human induced pluripotent stem cell-derived ovarian support cells as a clinical-grade product for in vitro fertilization [0.03%]
人诱导多能干细胞来源的卵巢支持细胞作为临床级别的体外受精产品的发展
Bruna Paulsen,Ferran Barrachina,Sabrina Piechota et al.
Bruna Paulsen et al.
Human induced pluripotent stem cells (hiPSCs) show promise in the development of novel strategies to alleviate reproductive diseases and improve reproductive outcomes. Here, we detail the clinical development and application of an ovarian s...
Retinal ganglion cell survival and functional maturation in transiently vascularized human retinal organoids [0.03%]
瞬时血管化的人视网膜类器官中的视网膜神经节细胞的存活和功能成熟
Kritika Sharma,Rouhollah Habibey,Mariana M Ribeiro et al.
Kritika Sharma et al.
Retinal organoids are widely used to model human retinal development and disease, but their utility is limited by the absence of vascular networks and stable axonal projections, which contribute to retinal ganglion cell degeneration and los...
Human pluripotent stem cell-derived innate and adaptive immune cells for cancer immunotherapy [0.03%]
人多能干细胞来源的先天性和适应性免疫细胞在癌症免疫治疗中的应用
Zahir Shah,Lei Tian,Michael A Caligiuri et al.
Zahir Shah et al.
Allogeneic cell-based therapies hold great promise for cancer immunotherapy but face challenges like scalability, immune rejection, graft-versus-host disease, and toxicities. Human pluripotent stem cells (hPSCs), including embryonic and ind...
A peripheral glial niche orchestrates the early stages of skin wound healing [0.03%]
一个外周神经胶质生态位协调皮肤伤口愈合的早期阶段
Salome Stierli,Adrian Salas-Bastos,Sofia Micheli et al.
Salome Stierli et al.
Skin repair is a complex, dynamic process involving multiple cell types. Using multiplex imaging, spatial transcriptomics, and single-cell RNA sequencing, we show that peripheral nerves-containing repair glia-form a pro-reparative niche clo...
Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells [0.03%]
纳米工程化三维培养基质能使基因工程造血干细胞具有优越的持久性和多系植入能力
Federico Midena,Laura Alessandrini,Claudio Conci et al.
Federico Midena et al.
Ex vivo culture of hematopoietic stem and progenitor cells (HSPCs) is required for gene therapy applications but inadvertently triggers detrimental cellular responses, potentially threatening clinical success. In this study, we employ nicho...