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Eficacia y seguridad de HSC CD34+ autólogas criopreservadas transducidas con un vector lentiviral EFS que codifica el gen ADA humano en sujetos ADA-SCID

Candidato terapéutico o dispositivo Células madre hematopoyéticas (HSC) CD34+ autólogas transducidas con un vector lentiviral que codifica el gen ADA humano (o ...
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Estudio de fase 1 de CD4LVFOXP3 autólogo en participantes con síndrome IPEX

El síndrome IPEX es una afección poco frecuente en la que el cuerpo no puede controlar ni restringir una respuesta inmunitaria, por lo que las células inmunitarias de la persona atacan su propio ...
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Proponemos ampliar y mejorar nuestro núcleo de células madre, un recurso clave para la zona. Este proyecto impulsará nuevas terapias y capacitará a científicos ...

Objetivo de la investigación Estudiamos cómo el cromosoma X inactivo se reactiva de forma aberrante en las células madre pluripotentes femeninas y desarrollamos formas de prevenirlo, lo que permite un ...
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Genetically Modified Hematopoietic Stem Cells for the Treatment of Danon Disease

Research Objective We propose to discover a novel, genetically modified hematopoietic stem cell based treatment for Danon disease, a rare lysosomal storage disease that affects the heart. Impact As ...
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Researcher Resources

CIRM provides funding across all stages of research, from basic science to translational studies and clinical trials. This support drives best-in-class research aimed at developing innovative ...
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CIRM Clinical Trials

Through its strategic funding of clinical trials, CIRM helps advance innovative stem cell and gene therapy discoveries toward future treatments for patients. CIRM funds clinical trials across a broad ...
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CIRM approves over $160M to support preclinical and clinical research, advances education opportunities and access to clinical trials

South San Francisco, CA, December 11, 2025 – The California Institute for Regenerative Medicine (CIRM) governing board today approved funding of over $160 million for 16 new research awards to develop ...
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Clinical Trial Stage Projects

The mission of California Institute for Regenerative Medicine (CIRM) is to accelerate stem cell treatments to patients with unmet medical needs. With our Clinical Stage Programs, we intend to speed up ...
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DISC5 Awards

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 ...
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癌症干细胞生物学中的 RNA 修饰。

细胞功能很大程度上取决于其基因表达，而基因表达调控着功能性蛋白质的合成。不同层面的机制控制着基因表达，以确保正确的蛋白质合成和细胞功能。其中一种控制机制涉及RNA分子的化学 ...
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CIRM awards $27 million to launch novel program designed to bring cell and gene therapies to underserved California communities

South San Francisco, Calif., October 30, 2025 – The California Institute for Regenerative Medicine (CIRM) has approved awarding $27 million through three grants across the state to create a network of ...
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CIRM approves $73 million in awards for discovery research

South San Francisco, CA, September 25, 2025 – The California Institute for Regenerative Medicine (CIRM) has approved awarding more than $73 million across 23 grants to support discovery research ...