Therapeutic/Technology: Therapeutic Approach
A drug-screening platform for autism spectrum disorders using human astrocytes
Autism spectrum disorders (ASD) are complex neurodevelopmental diseases that affect about 1% of children in the United States. Such diseases are mainly characterized by deficits in verbal communication, impaired social interaction, and limited and repetitive interests and behavior. The causes and best treatments remain uncertain. One of the major impediments to ASD research is the […]
Gene Targeting to Endogenous Stem Cells for Segmental Bone Fracture Healing
Segmental bone fractures are a complex medical condition. These injuries cause great suffering to patients, long-term hospitalization, repeated surgeries, loss of working days, and considerable costs to the health system. It is well known that bone grafts taken from the patient (autografts) are considered the gold-standard therapy for these bone defects. Yet these grafts are […]
Engineered iPSC for therapy of limb girdle muscular dystrophy type 2B
Limb girdle muscular dystrophy type 2B (LGMD 2B) is a form of muscular dystrophy that leads to muscle degeneration and disability. In LGMD 2B, a vital muscle protein is mutated, and its absence leads to progressive degeneration of muscles in the body that are needed for mobility. To create a therapy, we will provide a […]
Development of Novel Autophagy Inducers to Block the Progression of and Treat Amyotrophic Lateral Sclerosis (ALS) and Other Neurodegenerative Diseases
ALS is a progressive neurodegenerative disease that primarily affects motor neurons (MNs). It results in paralysis and loss of control of vital functions, such as breathing, leading to premature death. Life expectancy of ALS patients averages 2–5 years from diagnosis. About 5,600 people in the U.S. are diagnosed with ALS each year, and about 30,000 […]
Restoring vision by sheet transplants of retinal progenitors and retinal pigment epithelium (RPE) derived from human embryonic stem cells (hESCs)
There is currently no effective treatment to restore or improve vision for patients suffering from incurable blinding diseases such as dry age-related macular degeneration and retinitis pigmentosa, which need both new photoreceptors and retinal pigment epithelium. However, a unique method to transplant fetal retinal progenitor sheets together with its supporting retinal pigment epithelium (RPE) has […]
Preclinical and clinical testing of a stem cell-based combination product for insulin-dependent diabetes
Diabetes exacts a tremendous toll on patients, their families, and society. Autoimmune Type 1 diabetes, often called juvenile-onset diabetes, is caused by a person’s own immune system mistakenly destroying their insulin-producing cells in the pancreas, known as beta cells. When those beta cells are lost, the ability to produce insulin in response to consumed carbohydrates […]
In Utero Embryonic Stem Cell Transplantation to Treat Congenital Anomalies
Many fetuses with congenital blood stem cell disorders such as sickle cell disease or thalassemia are prenatally diagnosed early enough in pregnancy to be treated with stem cell transplantation. The main benefit to treating these diseases before birth is that the immature fetal immune system may accept transplanted cells without needing to use immunosuppressant drugs […]
Stem cell therapy for inflammatory bowel disease
One of the most promising approaches that physicians foresee for treating human disease is regenerative medicine. A major aim in this field is to restore function by repairing damaged organs. Inflammatory bowel disease (IBD) is a chronic disease characterized by intermittent episodes of intestinal inflammation and disruption of the intestinal epithelial barrier. It causes significant […]
Characterization of Human Skeletal Muscle Stem Cells for Clinical Application
Skeletal muscle makes up 40% of our bodies, dictates our form, is responsible for our ability to move, express ourselves, eat, breath and to look around. Restoration or preservation of the body’s normal form and function is the central goal of regenerative medicine and the central focus of my clinical specialty of plastic and reconstructive […]
Human endothelial reprogramming for hematopoietic stem cell therapy.
The current roadblocks to hematopoietic stem cell (HSC) therapies include the rarity of matched donors for bone marrow transplant, engraftment failures, common shortages of donated blood, and the inability to expand HSCs ex vivo in large numbers. These major obstacles would cease to exist if an extensive, bankable, inexhaustible, and patient-matched supply of blood were […]