The University of California and the British pharmaceutical giant GlaxoSmithKline on Thursday announced plans to build a new $67 million genetics laboratory focused on the gene-editing technology CRISPR.
Scientists at what will be called the Laboratory for Genomics Research, to be built over the next five years in San Francisco's Mission Bay, will explore how genetic mutations cause disease, while developing new gene therapies and other treatments.
The study of human genetics has exploded in the last decade, and scientists can now identify mutations in DNA that cause a wide range of disease, from cancer to Huntington’s to muscular dystrophy.
“But turning that into an actionable item where you can develop a therapy has been challenging,” said Jonathan Weissman, a biochemist at UCSF. Weissman is designing the laboratory with UC Berkeley's Jennifer Doudna, a CRISPR pioneer, and Hal Barron, chief science officer and president of GSK.
Weissman hopes the lab will enable researchers to more fully understand genetic differences through the advancement of functional genomics -- the study of gene relationships and interactions -- that relies on CRISPR technology.
CRISPR is so powerful because it targets specific genes with precise edits in DNA.
“CRISPR is a great discovery tool that lets us understand why changes in our DNA can cause disease and then give us clues as to how we might be able to intervene to prevent it,” said Weissman.
Recent advances in machine learning have given scientists the ability to use powerful computers to analyze the massive amount of data generated by CRISPR applications. The hope is that these supercomputers can help unlock the mysteries of cell biology and rapidly accelerate the discovery of new treatments.
“We think that human genetics, functional genomics and machine learning will allow us to identify novel targets that will result in medicines that will have a profound effect ,” said Barron.
GSK's Barron said he hopes the lab will spur advancements in gene therapy at “a pace previously thought impossible."
The lab will employ about 40 people, GSK and UC said, and will be located between UCSF’s Mission Bay campus and the new Warriors stadium.
CRISPR and Ethics
With CRISPR technology, scientists can modify or add entirely new genes.
CRISPR is different from other gene-editing tools in that its applications can alter the DNA of somatic cells and germ cells.
Somatic cells are found in organs and tissues and are not passed on through reproduction. Germ cells hold genes that are heritable.
Doudna said the focus of the new lab will be on fundamental discovery science, and not on germ cell editing.
“I don't think there's any intention right now to be editing human embryos in the center," she said. "I think our goal is actually to work on various kinds of disease-related questions that would be addressable using primary cells and tissues.”
Last year, controversy swarmed around He Jiankui, a biochemist with Southern University of Science and Technology in Shenzhen, China, who used CRISPR to perform germline editing to modify genes in a human embryo.
At the time, Doudna was one of the scientists who quickly criticized Jiankui, telling NPR that his work is a “break from the cautious and transparent approach of the global scientific community’s application of CRISPR-Cas9 for human germline editing.”
Doudna has declared a need to confine the use of gene-editing in human embryos to situations in which there is a clear medical need with zero alternative viable approach.
Additionally, Doudna says there’s a lot of fundamental research that needs to be done prior to any use of genome editing for clinical purposes in human embryos. “In that regard, the [lab] will play a very important role in stimulating fundamental, curiosity-driven research that needs to be done,” she said. “It will both advance our understanding of the human genome and we’ll also advance the potential in the power of the technology."