Developing new therapies for neurodegenerative diseases

Current projects in our lab center on the following 4 areas:

  1. iPSC modeling of neurodegenerative diseases and biomarker development: We used human iPSC from patients and healthy controls and differentiate them into neurons, microglia and other cell types relevant to the CNS. We aim to build better models of human disease (including Alzheimer’s disease, FTD and ALS) that replicate the pathobiology of human patients and better predict patient response to therapies we are developing. We build biomarker assays in human cells and tissues that will facilitate future clinical trials.
  2. CRISPR gene editing of mutations that cause neurodegenerative diseases, including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). Our group is currently focused on correcting single gene mutations that cause FTD and ALS using novel CRISPR approaches. We are building the tools necessary to advance these discoveries to clinical trials.
  3. Delivery of CRISPR technology to human CNS cell types: One of the major barriers to utilizing CRISPR as a therapy for neurologic disease is delivery of CRISPR technology to targeted cells and tissues. This challenge is amplified by biological sequestration of the CNS (such as through the blood brain barrier) – the brain actively defends against bacterial invaders! Combining clinical expertise and basic biology, we have developed screening platforms to test CRISPR delivery technologies in relevant human cell types
  4. Bioinformatic tools to faciliate gene therapy and future clinical trials.