Webinar: A CRISPR Focus on Editing by LabX Media Group

Host Login

About This Webinar

There is no denying the impact of CRISPR-Cas9 technology
as a molecular tool for understanding basic biology, but
recent advances push the gene-editing platform from analytic
resource to therapeutic solution. This webinar will present
efforts to address the challenges of genome modification as
therapy, whether by improving fidelity, introducing fail-safes, or
working ex vivo.

Agenda

Next generation CRISPR technologies for potential clinical use
Developing gene-based therapies for metabolic and neurodegenerative diseases

Presenters

Natalia Gomez-Ospina, MD, PhD

Taube Pediatric Neurodegenerative Disease Scholar Assistant Professor, Dept. of Pediatrics Divisions of Medical Genetics & Stem Cell and Regenerative Medicine Stanford University

Dr. Gomez-Ospina is currently an Assistant Professor in the Department of Pediatrics. She is a board-certified medical geneticist who is interested in the development of definitive therapies for individuals with genetic diseases. She leads a Program for Inherited Metabolic Diseases at Stanford that aims to promote the development of gene and cell-based therapies for metabolic and neurodegenerative diseases at Stanford. She has been the lead author in research studies in The New England Journal of Medicine, Cell, Nature Communications, and American Journal of Medical Genetics.

Her recent work has focused on developing therapeutic genome of hematopoietic stem cells as a therapeutic approach for neurodegenerative diseases. She established a flexible, “one-size-fits-many” genetic approach that is independent of specific genes and mutations. This approach, in which she commandeers the hematopoietic system to express and deliver therapeutic proteins to the brain can improve the treatment of neurological diseases beyond LSDs and has been shown to effective in a preclinical model of Mucopolysaccharidosis type I (MPSI or Hurler syndrome) and in Gaucher disease.

Pranam Chatterjee, PhD

Carlos M. Varsavsky Research Fellow Harvard Medical School | MIT Media Lab

Pranam Chatterjee is a current research fellow at Harvard Medical School and a recent PhD graduate of the MIT Media Lab, where he engineered novel CRISPR tools for broad biological applications, utilizing both computational and experimental methodologies. He has published seminal work on the discovery and enhancement of CRISPR-Cas9 enzymes for broad, efficient, and specific genome editing. These novel enzymes have been employed to edit the genomes of diverse organisms, from rice to rabbits, and are further being developed to treat genetic diseases, such as Huntington’s Disease and Rett Syndrome. Additionally, he has extensive experience in cancer immunotherapy, having co-authored foundational work on the functional mechanisms of PD-1 checkpoint blockade. Finally, Pranam’s established expertise in bioinformatics and machine learning are currently being applied for the development of in vitro gametogenesis protocols.