Patients with end-stage heart failure (ESHF) have a 2-year survival rate of 50% with conventional medical therapy. This dismal survival rate is actually significantly worse than patients with AIDS, liver…
The primary goal is to bring a safe and effective therapy to children with severe large airway disease. Our intent is to implement all of the necessary steps for a…
Science has made great progress in the treatment of certain cancers with targeted and combination therapies, yet prolonged remissions or cures are rare because most cancer therapies only inhibit cell…
A stroke kills brain cells by interrupting blood flow. The most common “ischemic stroke” is due to blockage in blood flow from a clot or narrowing in an artery. Brain…
Amyotrophic lateral sclerosis (ALS), a lethal disease lacking effective treatments, is characterized by the loss of upper and lower motor neurons. 5-10% of ALS is familial, but the majority of…
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…
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…
Diabetes exacts a tremendous toll on patients, their families, and society in general. Autoimmune Type 1 diabetes, often called juvenile-onset diabetes, is caused by a person’s own immune system mistakenly…
The adult human heart contains small numbers of cardiac stem cells that are able to partially repair the heart following a heart attack or throughout the course of progressive heart…
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