Gene Therapy Initiative
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Leadership
Meet Stephanie Cherqui, PhD
Dr. Cherqui focuses on developing stem cell and gene therapy strategies for degenerative multi-systemic disorders such as cystinosis, and to understand the molecular mechanisms by which hematopoietic stem cell progeny can lead to tissue repair in non-hematopoietic genetic diseases.
Meet Alysson Muotri, PhD
Dr. Muotri's research is focused on solving one of life's greatest mysteries: What is it that makes us uniquely human? He is studying the brain from an evolutionary and developmental perspective, differentiating stem cells to recreate "brain organoids" in the controlled setting of a lab.
Meet Betty Cabrera, MPH
Betty joins UCSD's GTI as Director of Engagement and Operations, with over a decade of experience in gene therapy development. Her role involves leveraging extensive knowledge and network in regenerative medicine, clinical research, and patient interaction, to create a leading gene therapy program at UCSD.