Mara Pavel-Dinu PhD

Seattle Children’s Research Institute
Assistant Professor, Pediatrics

Email: mara.pavel-dinu@seattlechildrens.org | Phone: 206.884.4704

In Pavel-Dinu Lab, we combine regenerative biology with CRISPR/Cas9-based genome editing technology to rewrite the DNA code of a defective gene, restore its function, and re-establish normal hematopoiesis. Our genome editing platform can effectively and precisely modify the DNA sequence, at nucleotide resolution, in hematopoietic stem cells (HSCs) cultured outside the body (ex vivo) to restore the immune development and function following transplantation (in vivo). This targeted therapeutic approach has revolutionized medicine for rare blood diseases (e.g., sickle cell disease). However, patients with rare mono-genetic immune diseases, collectively referred to as Inborn Errors of Immunity (IEI), have yet to benefit from it.

The study of human hematopoietic regeneration is hampered by the limited number of HSCs in our bodies, the inability to expand these rare cells outside the body, and, for ethical reasons, using patients’ cells for research purposes only.  IEIs are a growing class of rare mono-genetic immune diseases that add great value to our scientific endeavors and have vast implications for advancing medicine.  From the root cause to the molecular mechanism and disease progression, IEIs offer the complete disease layout for us to study the complex biological processes of hematopoietic regeneration, uncover new principles, and transform how we approach medicine in the 21st century.

Our research mission is to discover the genetic, immune, metabolic, and transcriptional networks that orchestrate hematopoietic regeneration, understand how it goes awry in IEIs, and turn our scientific insights into innovative and long-lasting gene and cell therapies accessible for an ethnically and age-diverse community of patients.