IGI research centers around genome editing, incorporating researchers focused on human health applications, agricultural applications, development of genome-editing technology, and translation of lab discoveries into real-world solutions.
Advancing genome engineering Since its founding, IGI researchers have discovered multiple new genome-editing proteins, expanding the toolkit beyond
Cas9. The wave of discoveries of additional genome-editing tools with different properties, including new Cas proteins and techniques like
base editing, was sometimes called "CRISPR 2.0" in popular science reporting. Ultra-compact proteins CasX and CasY were discovered by Jillian Banfield and collaborators at the IGI in some of the world's smallest microbes. Another compact Cas protein, CasΦ ("Cas phi"), was discovered by Banfield and Doudna and colleagues in the genomes of huge
bacteriophages. Doudna and other IGI researchers have also advanced new techniques to improve non-viral and
in vivo delivery of CRISPR-based therapeutics for medical applications, and worked on improving CRISPR safety and precision.
Human health The IGI human health program has focused on developing therapies for rare and neglected genetic diseases and platform technology approaches to addressing rare diseases, including
sickle cell disease and other blood and immune disorders. In 2021, the US Food and Drug Administration approved a
clinical trial for an experimental CRISPR-based therapy for sickle cell disease developed by a consortium including the IGI,
UCSF Benioff Children's Hospital, and the UCLA Broad Stem Cell Research Center. Other health research at the IGI focuses on
cancer,
neurodegenerative diseases, and
clinical diagnostics. In 2024, the IGI worked with the
Children's Hospital of Philadelphia to develop a personalized CRISPR therapy for a baby born with a rare fatal genetic disease affecting the
urea cycle, which was approved and delivered to the patient in just six months. This event marked the first use of in vivo gene editing to repair a gene.
Climate and sustainable agriculture The IGI
sustainable agriculture program and its Plant Genomics and Transformation Facility has developed CRISPR protocols for editing over 30 common crop species, removing toxic cyanide precursors in
cassava, and improving
drought tolerance in rice. In 2022, the IGI launched new programs to apply genome editing and genomic technologies to the challenge of mitigating and adapting to
climate change. This work included efforts to reduce agricultural emissions, capture atmospheric carbon, and help farmers adapt to changing conditions. The
Chan Zuckerberg Initiative committed $11 million to the IGI to support research on CRISPR-based approaches to enhancing the ability of plants and soils to remove and sequester atmospheric carbon. At the 2023
TED conference in Vancouver, it was announced that the IGI was selected for funding by the
Audacious Project and the institute received $70 million from donors to develop
microbiome editing tools that can be applied to real-world problems related to human health and climate change. The project, entitled "Engineering the Microbiome with CRISPR to Improve our Climate and Health," is initially targeting two problems caused by microbiomes,
methane emissions from livestock, and
childhood asthma. In a meeting with US senators in December 2018, Doudna was asked about the potential high cost of a CRISPR-based treatment of sickle cell disease and what could be done to bring these costs down. When she returned to the IGI following this meeting, she decided to make affordability a part of the mission of the IGI, and a key goal for its sickle cell initiative. == CRISPR education ==