Molecules to Correct Aberrant RNA Signature in Human Diseased Neurons
Approximately 5,600 people in the U.S. are diagnosed with ALS each year. The incidence of ALS is two per 100,000 people, and it is estimated that as many as 30,000…
Use of human iPS cells to study spinal muscular atrophy
Spinal muscular atrophy (SMA) is one of the most common autosomal recessive disorders that cause infant mortality. SMA is caused by loss of the Survival of Motor Neuron (SMN) protein,…
Correlated time-lapse imaging and single cell molecular analysis of human embryo development
We understand little about human development especially at the earliest stages. Yet human developmental biology is very important to stem cell biology and regenerative medicine for two reasons: 1) Understanding…
Discovery of mechanisms that control epigenetic states in human reprogramming and pluripotent cells
The CIRM Basic Biology Award III was developed to support basic research that enables the realization of the full potential of human stem cells and reprogrammed cells for therapies and…
Functional characterization of mutational load in nuclear reprogramming and differentiation
One of the most potentially powerful aspects of regenerative medicine is stem cell therapy. In this therapy, healthy tissues derived from stem cells will be implanted into patients with damaged…
Triplet Repeat Instability in Human iPSCs
Over twenty human genetic diseases are caused by expansion of simple DNA sequences composed of repeats of three nucleotides (such as CAG, CTG, CGG and GAA) within essential genes. These…
Neural and general splicing factors control self-renewal, neural survival and differentiation
Human embryonic and patient-specific induced pluripotent stem cells have the remarkable capacity to differentiate into many cell-types, including neurons, thus enabling the modeling of human neurological diseases in vitro, and…
Elucidating Molecular Basis of Hypertrophic Cardiomyopathy with Human Induced Pluripotent Stem Cells
Familial hypertrophic cardiomyopathy (HCM) is the leading cause of sudden cardiac death in young people, including trained athletes, and is the most common inherited heart defect. Until now, studies in…
Generation and characterization of corticospinal neurons from human embryonic stem cells
A major goal of stem cell research is to generate various functional human cell types that can be used to better understand how these cells work and to use them…
Molecular Mechanisms Underlying Human Cardiac Cell Junction Maturation and Disease Using Human iPSC
Heart disease is the number one cause of death and disability in California and in the United States. Especially devastating is Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), an inherited form of…
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