ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS
Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry…
Human Cardiovascular Progenitors, their Niches and Control of Self-renewal and Cell Fate
For the millions of Americans who are born with or develop heart disease, stem cell research offers the first hope of reversing or repairing heart muscle damage. Thus, early reports…
Innate Immune Regulation of Lung Alveolar Stem Cell Renewal in Mouse and Man
The lung, along with the skin and gut are the three organs in perpetual contact with our environment. The lung has evolved mechanisms for repairing injury due to exogenous noxious…
A novel druggable mechanism to safeguard stem cell genome
Safeguarding the genome is essential for cells’ proper functions, and more importantly for safe and efficacious applications involving pluripotent stem cells and adult stem cells. However, how pluripotent or somatic…
Molecular basis of plasma membrane characteristics reflecting stem cell fate potential
Stem cells generate mature, functional cells after proteins on the cell surface interact with cues from the environment encountered during development or after transplantation. Thus, these cell surface proteins are…
New Regulators of Spermatogonial Stem Cells: RHOX Homeobox Transcription Factors
Infertility afflicts a remarkably high percentage (~15%) of couples, with male factor defects being responsible for more than ½ of these cases. One-third of these male infertility cases have no…
Differentiation of Human Hematopoietic Stem Cells into iNKT Cells
Blood stem cells living in the bone marrow of adult humans give rise to all of the cells in our blood, including the red blood cells that carry oxygen to…
Tissue Engineered Recellularized Laryngotracheal Implants
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…
Clinical Trial of Stem Cell Gene Therapy for Sickle Cell Disease
Sickle cell disease (SCD)results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen. It occurs with a frequency of…
A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells
β-thalassemia is a genetic disease caused by diverse mutations of the β-globin gene that lead to profoundly reduced red blood cell (RBC) development. The unmet medical need in transfusion-dependent β-thalassemia…