Our proposal details assembly of a multidisciplinary Disease Team whose goal will be to take into the clinic a novel and promising stem cell-based strategy for selectively targeting invasive tumor…
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to “sickle” under conditions of low oxygen, occurs with a frequency…
Huntington’s disease (HD) is a devastating degenerative brain disease with a 1 in 10,000 risk of having a mutation that inevitably leads to death. These numbers do not fully reflect…
Children born with sickle cell anemia (SCA), caused by a genetic defect in hemoglobin, have severe anemia and damage to virtually all the body organs: the damage begins in infancy,…
The present planning grant application lays the groundwork for a collaborative heart disease regenerative medicine team. We plan to develop sequential preclinical and clinical investigations directed at regenerative stem cell-based…
Brain tumors (BTs) are incurable, whether they start in the brain or spread there from other sites (e.g., lung, colon, breast, skin). Indeed, the latter situation is even more common…
The targeted disease is retinitis pigmentosa (RP), a severe form of blindness that runs in families. This disease is not common, yet represents an attractive near term target for stem…
Parkinson’s disease (PD) is a devastating movement disorder caused by the death of dopaminergic neurons (a type of neurons in the central nervous system) present in the midbrain. These neurons…
RNA interference is a naturally occurring means to block the function of genes in our body. We propose that RNA interference can be used to block HIV-1 infection and its…
Alzheimer’s Disease (AD) is a major health problem in the U.S. with 10% of people over the age of 65 and 50% of people over the age of 85 afflicted…
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