Disease Focus: Genetic Disorder


Mechanisms to maintain the self-renewal and genetic stability of human embryonic stem cells

Human embryonic stem cells (hESCs) are capable of unlimited self-renewal, a process to reproduce self, and retain the ability to differentiate into all cell types in the body. Therefore, hESCs hold great promise for human cell and tissue replacement therapy. Because DNA damage occurs during normal cellular proliferation and can cause DNA mutations leading to […]

Molecular and Cellular Transitions from ES Cells to Mature Functioning Human Neurons

Human embryonic stem cells (hESCs) are pluripotent entities, capable of generating a whole-body spectrum of distinct cell types. We have developmental procedures for inducing hESCs to develop into pure populations of human neural stem cells (hNS), a step required for generating authentic mature human neurons. Several protocols have currently been developed to differentiate hESCs to […]

Using Human Embryonic Stem Cells to Understand and to Develop New Therapies for Alzheimer’s Disease

Alzheimer’s Disease (AD) is a progressive incurable disease that robs people of their memory and ability to think and reason. It is emotionally, and sometimes financially devastating to families that must cope when a parent or spouse develops AD. Unfortunately, however, we currently lack an understanding of Alzheimer’s Disease (AD) that is sufficient to drive […]

Functional Genomic Analysis of Chemically Defined Human Embryonic Stem Cells

Regenerative medicine holds the promise that tissues can be engineered in vitro and then transplanted into patients to treat debilitating diseases. Human Embryonic Stem Cells differentiate into a wide array of adult tissue types and are thought to be the best hope for future regenerative therapies. This grant has three main goals: 1. The creation […]

Derivation and characterization of human ES cells from FSHD embryos

Facioscapulohumeral muscular dystrophy (FSHD) is the third most common hereditary muscular dystrophy. It is autosomal dominant, meaning that if one of the parents has the disease, their children have a 50:50 chance of getting it, too. FSHD is characterized by progressive weakness and atrophy of facial, shoulder and upper arm musculature, which can spread to […]

Molecular Characterization of hESC and hIPSC-Derived Spinal Motor Neurons

One of the main objectives of stem cell biology is to create physiologically relevant cell types that can be used to either facilitate the study of or directly treat human disease. Tremendous progress towards these goals has been made in the area of motor neuron disease and spinal cord injury through the findings that motor […]

Stem Cells: Fulfilling the Promise – 2011 CIRM Grantee Meeting

*Please note: this video contains graphic images of a disfiguring disease John Wagner presented the closing scientific talk at the 2011 CIRM Grantee Meeting. Weaving together stories of medical history, literature, bioethics, and his own clinical work, Wagner describes the challenges and promise of stem cell based therapies. Dr. Wagner is a professor of pediatrics, […]

Spotlight on Disease Team Awards – Genetic Skin Disease: Welcoming Remarks

Spotlight on Disease Team Awards – Genetic Skin Disease: Welcoming Remarks Robert Klein J.D., gave the welcoming remarks for the “Spotlight on Disease Team Awards: Genetic Skin Disease,” an educational event presented at the CIRM Governing Board meeting on February 4, 2010. Klein is chair of the CIRM Governing Board. The CIRM-hosted event was presented […]

Spotlight on Disease Team Awards – Genetic Skin Disease: Introduction

Spotlight on Disease Team Awards – Genetic Skin Disease: Introduction Phillip Pizzo, M.D., spoke at the “Spotlight on Disease Team Awards: Genetic Skin Disease,” an educational event presented at the CIRM Governing Board meeting on February 4, 2010. Pizzo is dean of the Stanford School of Medicine and is a member of the CIRM Governing […]

Spotlight on Disease Team Awards – Genetic Skin Disease: Lynn Anderson

Spotlight on Disease Team Awards – Genetic Skin Disease: Lynn Anderson Lynn Anderson, spoke at the “Spotlight on Disease Team Awards: Genetic Skin Disease,” an educational event presented at the CIRM Governing Board meeting on February 4, 2010. Anderson is President and Founder of the Epidermolysis Bullosa Research Foundation. She was introduced by Albert Lane, […]