A hNSC Development Candidate for Huntington’s Disease
Huntington’s disease (HD) is a devastating degenerative brain disease with at least a 1 in 10,000 prevalence that inevitably leads to death. These numbers do not fully reflect the large…
Huntington’s disease (HD) is a devastating degenerative brain disease with at least a 1 in 10,000 prevalence that inevitably leads to death. These numbers do not fully reflect the large…
Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…
Myelomeningocele – also known as spina bifida – is a devastating and costly defect that causes lifelong paralysis as well as bowel and bladder incontinence in newborns. It is one…
Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been…
We are developing a stem cell-derived replacement cell therapy for insulin-requiring diabetes. Through a process known as directed differentiation, embryonic stem cells are turned into pancreatic cells in the laboratory.…
The proposed project is designed to assess the safety and preliminary activity of escalating doses of human embryonic stem cell derived oligodendrocyte progenitor cells (OPCs) for the treatment of spinal…
The HIV-1 virus enters cells by binding to a protein called CCR5 on the cell surface. A naturally occurring mutation in CCR5, CCR5d32, has been shown to provide protection from…
It is estimated that by 2020, over 450,000 Californians will suffer from vision loss or blindness due to the age-related macular degeneration (AMD), the most common cause of retinal degeneration…
The goal is to bring a safe and effective therapy to adult patients with critical narrowing of the upper windpipe (trachea) and lower voicebox (larynx). Our intent is to implement…
Cancer is a major cause of morbidity and mortality worldwide. Many believe that progress in drug development has not been as rapid as one would have predicted based on the…