The mission of the California Institute for Regenerative Medicine (CIRM) is to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a ...

Severe Combined Immunodeficiency (SCID) is a rare primary immunodeficiency in which patients lack T and B cells, the white blood cells needed to fight infections. Without a bone marrow stem cell ...

The mission of the California Institute for Regenerative Medicine (CIRM) is to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a ...

The mission of California Institute for Regenerative Medicine (CIRM) is to accelerate world class science to deliver transformative regenerative medicine treatments in an equitable manner to a diverse ...

Neuropsychiatric disorders correlate to impaired metabolism, though understudied. We will describe how metabolism impacts disorders etiology and identify new, tractable therapeutic strategies. We will ...

基因治疗产品将恢复 DOK7 基因的功能性副本，从而产生正常的 DOK7 蛋白质，从而重建 NMJ 中的正确信号传导，最终治愈 DOK7 CMS。 未满足的医疗需求 DOK7 CMS 目前尚无治愈方法。拟议的基因疗法将是 ...

壁细胞对于健康成熟脉管系统的发育和维持是不可或缺的，对于血管治疗和发育模型有价值。 然而，它们的功能可塑性、发育多样性和多种分化途径使体外生成变得复杂。 幸运的是，体内研究 ...

Chromosomal microdeletion syndromes result in severe neuropsychiatric syndromes and lack therapy. This proposal will define critical genomic regions needed to generate new tools for functional rescue.

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染色体微缺失综合征会导致严重的神经精神综合征，且缺乏治疗。本提案将定义关键基因组区域，以开发新的功能挽救工具。 冲击 这项研究为恢复16p和22q缺失综合征的基因功能提供了一种新方法。

Thassio Mesquita, Rodrigo Miguel-Dos-Santos, Weixin Liu, Mario Fournier, Russell G Rogers, Jocelyn Alfaro, Asma Nawaz, Lizbeth Sanchez, Xaviar M Jones, Liang Li, Eduardo Marban, Eugenio Cingolani ...

许多组织，例如成年人的心脏，在损伤后无法充分再生。(2,3) 组织工程策略提出了帮助身体恢复和修复的创新。例如，TE 方法可能能够减弱心肌梗塞 (MI) 后的心脏重塑，并可能将总体心脏功能 ...