Organoids were cultured from healthy donor iPSC sources and differentiated using the human iPSC-derived Cerebral Organoid ...

1 Indeed, last November the FDA approved PTC Therapeutics’ Kebilidi, the first U.S. gene therapy to be administered directly into the brain. 2 The AAV-based therapeutic treats a rare fatal ...

AAV-Navβ1 gene therapy effectively reduces seizures and prolongs life in a mouse model of SCN1B-linked Dravet syndrome, ...

脂肪组织与中枢神经系统之间存在复杂的通信机制，以维持全身能量稳态。传统观点认为，脂肪组织通过分泌循环激素向大脑传递代谢状态信息，而大脑则通过交感神经输出调节脂肪细胞功能。背根神经节（DRG）的感觉神经元也被证实支配脂肪组织，但其生理重要性因缺乏特异性 ...

Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form the basis for several approved gene therapies for human diseases, mainly owing to their ability to sustain ...

Feb. 11, 2025 — Scientists have developed an innovative method for growing brain tumors of individual patients in the laboratory that mimic the original structure and the molecular property of ...

was systemically administered to mice with AAV neutralizing antibodies, the gene transfer efficiency in the brain and liver decreased to 5–15%. However, by loading AAV9 into the nanomachine ...

TFE3 overexpression attenuates α-Syn toxicity in a mouse model of Parkinson's disease. TFE3 overexpression rescues autophagy defects of dop ...