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Biomedicine

© 2017 Barbara Ries

Mutations in single genes cause more than 5000 human diseases.

Doctors can only treat the symptoms of genetic disease and patients often need to take non-curative drugs for their entire life. Scientists have known the cause of many genetic disorders for years, but until now there has been no clear way to cure them. The IGI aims to use the ‘molecular scalpel’ Cas9 to permanently correct disease-causing mutations in cells, animal models, and eventually human patients.

15

Principal Investigators

12

Research Projects

16

Published Papers

Hand holding slides with sections of tissue. © Noah Berger 2016

Key Goals

Work to understand the genetic basis of complex diseases
Identify new disease-modifying genes
Find ways to prevent disease
Develop CRISPR-based therapies for diseases that affect underserved populations

Biomedicine Projects