Liver transplantation (LT) has been used to treat a variety of liver diseases. Within hours after birth, neonates can present with disorders of the urea cycle (UCDs), the critical metabolic…
In ADA-SCID, allogeneic hematopoietic (blood) stem cell transplants from non-matched sibling donors are a high risk procedure. Additionally, the efficacy of chronic enzyme replacement therapy is uncertain in the long-term. A team at…
Research Objective A complete methodology for high efficiency, minimal toxicity hematopoietic stem cell transplantation for treatment of pediatric neurodegenerative disorders. Impact Improve treatment and outcomes for patients with neurodegenerative disorders…
Research Objective We will develop genome editing tools to identify silencers that regulate neural stem cell fate, uncovering key DNA elements that guide neurodevelopment and are disrupted in neurodevelopmental diseases…
Research Objective We develop a powerful platform to correct gene expression defects caused by haploinsufficiency and will show its ability to identify novel targets using iPSC-derived neurons and cortical organoids.…
