Development of a cell and gene based therapy for hemophilia
Hemophilia B is a bleeding disorder caused by the lack of FIX in the plasma and affects 1/30,000 males. Patients suffer from recurrent bleeds in soft tissues leading to physical…
Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke
The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct). Stroke is the third leading cause of death in…
Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke
The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct). Stroke is the third leading cause of death in…
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…
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.…
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,…
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.…
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…
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…
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…
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