It takes a lot of effort to develop a promising stem cell research idea into an effective treatment that can help patients. CIRM funds a pipeline of projects spanning discovery, translation and ...

Objetivo de la investigación Se combinará un injerto de pasta articular que contiene células madre mesenquimales y un hidrogel adhesivo que favorece el crecimiento del cartílago para lograr un ...

Sphingosine phosphate lyase insufficiency syndrome (SPLIS) is a rare genetic condition caused by mutations in the SGPL1 gene. This gene encodes an essential enzyme needed to metabolize a group of ...

The Sanford Burnham Prebys Institute’s third cohort of Summer Program to Accelerate Regenerative medicine Knowledge (SPARK) interns were comprised of young scholars representing the diversity of ...

Nous visons à découvrir une thérapie génique CRISPRi virale adéno-associée (AAV) pour les patients atteints de sclérose latérale amyotrophique (SLA) présentant des expansions répétées ...

This meeting of the Application Review Subcommittee will be conducted in person and via noticed teleconference locations, which will be made accessible to the public. Members of the public may attend ...

KIF1A-associated neurological disorder (KAND) is a neurodegenerative and often lethal ultrarare disease with a wide phenotypic spectrum associated with largely heterozygous de novo missense variants ...

Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This ...

beta-globin lentiviral vectors (beta-LV) have faced challenges in clinical translation for gene therapy of sickle cell disease (SCD) due to low titer and sub-optimal gene transfer to hematopoietic ...

Metabolism regulates cell fates during early mammalian cell differentiation. This protocol describes the steps for directed differentiation of primed human pluripotent stem cells (hPSCs) into the ...

We previously showed that inhibition of glycolysis in cutaneous squamous cell carcinoma (SCC)-initiating cells had no effect on tumorigenesis, despite the perceived requirement of the Warburg effect, ...

Colin M McCrimmon, Daniel Toker, Marie Pahos, Kevin Lozano, Jack J Lin, Jack Parent, Andrew Tidball, Jie Zheng, Laszlo Molnar, Istvan Mody, Bennett G Novitch, Ranmal A Samarasinghe Neurodevelopmental ...