Muscular dystrophies are a group of degenerative diseases that arise from genetic mutations that cause a loss of function in the corresponding gene products. Of particular interest is Limb Girdle Muscular Dystrophy Type 2B (LGMD2B), which is caused by mutations in the Dysferlin gene (DYSF). Here, we describe a potential gene therapy approach to deliver the healthy, full-length DYSF coding sequence in a safe and long-lasting manner to muscle fibers. We have injected Dysferlin-negative mice with naked DNA designed to knock-in DYSF using φC31 integrase.

Pluripotent stem cells provide a promising source to derive endothelial progenitor cells (EPs) for the treatment of vascular disease, ischemia, and stroke. We have developed a robust protocol to differentiate human iPSCs into nearly pure EP population (94-97% CD31+ and 78-83% VE-Cadherin+). These EPs also expressed other endothelial markers, vWF and NOS3. Compared to HUVECs, these EPs had higher level of artery marker, HEY1, and lower level of venous marker, Coup-TFII.

Peripheral arterial disease is an ischemic tissue related disorder characterized by a lack of adequate blood flow to the extremities. This occurs when the artery is blocked, typically as a result of atherosclerosis. Mesenchymal stem cells (MSCs) have been shown to be a possible therapeutic for ischemic diseases through their ability to secrete pro-angiogenic signals. Recently, it was shown that exosomes isolated from these MSC are key components in the delivery of pro-angiogenic factors.

Neurogenesis is the process of generating new neurons from undifferentiated neural stem and progenitor cells (NSPCs). Currently, the functional role of the NSPCs in the subgranular zone (SGZ) is largely attributed to the new neurons that NSPCs create. However, Recent study showed the hippocampal progenitors secrete large quantities of the essential growth factor VEGF in vitro and vivo. These findings arise the possibility that NSPCs could be an unexpected source of VEGF in the brain. Interestingly, after a seizure, the VEGF levels highly increase.

Neural Crest Stem Cells (NCSC) are a multipotent population of stem cells that arise from the neural plate border and possess the ability to migrate ventrally throughout the developing embryo. NCSCs retain multipotency prior to migration, during, and after migration, and are capable of differentiating into a variety of cells including cartilage, bone, melanocytes, autonomic neurons, and Schwann cells among others. During development cranial NCSCs migrate into the first and second pharyngeal arch and differentiate into the bones and cartilage that make up the craniofacial structure.

In order to study disease in physiologically relevant cell models, it is important to develop technologies that enable efficient control of gene expression. Using the newly developed CRISPR interference (CRISPRi) technology, we aim to interrogate genes involved in iPSC growth and maintenance at the whole-genome level. CRISPRi utilizes a deactivated Cas9 enzyme with a fused KRABs domain (dCas9-KRAB) for gene silencing. We have generated a genome-wide kinase and phosphatase library of 13,000 single guide RNAs designed to target 2600 genes.

The Petritsch lab at UCSF investigates many processes of one of the most important organ in the body, the brain. One of your main goals is to understand the development and maturation of myelinating cells called oligodendrocytes (OLG). OLG cells constituting 2-9% of the total cells in the adult brain are the largest proliferating population. These unique cells originate from oligodendrocyte precursor cells (OPC) that express the NG2 chondroitin sulfate proteoglycan.

The Calos lab is focused on creating therapies for the treatment of various muscular dystrophies. Muscular dystrophies are a collection of diseases characterized by increasing muscle weakness and wasting as the affected individual gets older. The most progressive and lethal of these myopathies is Duchenne Muscular Dystrophy, DMD is the result of a mutation in the dystrophin gene. This disease is sex linked, affects 1 in 3500 live male births and causes wasting to the muscles of the skeletal and cardiac systems.

The Fan Yang Laboratory focuses in Biomaterials and Stem Cells engineering for tissue engineering and targeted drug delivery. One of the major focuses of the lab is the eradication of glioblastoma multiforme (GBM) using adipose derived stem cells (ADSCs). Due to the delicate anatomical location of GBM (the brain), surgical removal is often times precluded, dramatically reducing clinical outcomes. Adipose derived stem cells are an abundant source of adult stem cells that possess unique tumor-tropic abilities coordinated by a myriad of tumor-secreted signals.

Psoriasis is the most common inflammatory skin disease affecting 1-3% of the world’s population. Both keratinocyte cells and immune cells are central in the disease pathogenesis. The Marinkovich Lab at Stanford University has established that psoriatic keratinocytes have an intrinsic predisposition for Rac1 hyperactivation. Rac1 is a small GTPase known to regulate many important cellular processes, including those mediating cell cytoskeletal rearrangements, cellular migration, cell-cell adhesion, and the cell cycle.