Human embryonic stem cells (hESC) have the remarkable capacity to replicate indefinitely and differentiate into virtually any cell type in the human body. Maintaining this pluripotent cell state requires the…
With their ability to develop into virtually all mature cell types, human pluripotent stem cells (hPSC) represent a unique and powerful research tool to study the fundamental mechanisms regulating human…
Stem cell quality and safety in developing regenerative medicine therapies is of utmost importance. Poor outcomes include inadequate functionality, exhaustion, immune rejection, cancer development, and others. Recent studies strongly support…
Pluripotent stem cell research is just on the verge of beginning to fulfill its promise to revolutionize medicine. Whether they are derived from embryos, or from adult cells that have…
One of the main objectives of stem cell biology is to create physiologically relevant cell types that can be used to either facilitate the study of or directly treat human…
The development of methods to “reprogram” adult cells such as skin cells by simultaneously expressing four specific factors — Oct3/4, Sox2, c-Myc and Klf4 — in order to create cells…
Stem cell biology and its applications to cell-based therapies, since its inception 30 years ago, has been hindered by the immunological considerations of rejection of non-autologous cells in patients, as…
Research Objective Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…
Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…
Research Objective To discover CRISPR-based genetic strategies that will directly and therapeutically modify cardiac myosin heavy chain isoform expression to treat HF and cardiomyopathies. Impact Heart Failure and Cardiomyopathies Major…
