University of Wisconsin biologist James Thomson and his lab attained the first laboratory-made human embryonic stem cells 20 years ago. Ever since, researchers have been building on this discovery, progressing the world’s knowledge of cellular biology and its potential in treating diseases.
Halfway through its third year in operation, the UW Human Stem Cell Gene Editing Service has strived to further stem cell research by providing the unique service of genetically engineering induced pluripotent stem cells with the newest gene-editing tool, the CRISPR-Cas9. These IPS cells are used for an array of interdisciplinary research projects at UW. The Human Stem Cell Gene Editing Service generates cell lines so labs can focus on addressing underlying scientific questions in their varying areas of interest.
Demand for these services has already prompted an expansion of the program after the UW2020 funding, which jump-started the program, stopped in 2017. Now, they are contemplating another expansion due to increasingly high demand.
IPS cells are master stem cells that can form any cell type present in the body. Like the first embryonic stem cells discovered on campus in 1998, IPS cells can be grown indefinitely in culture, and they can also be formed into various cell types.
Tim Kamp, director of the UW Stem Cell and Regenerative Medicine Center, said IPS cells are different because they are not obtained from in vitro fertilization embryos that were donated for research. Instead, they are obtained through technology.
“I could take a little piece of skin or a tube of blood from you and make a master stem cell called an induced pluripotent stem cell that is genetically identical to you and can form cell types that are genetically identical to the cells in your heart, brain, skeletal muscle, pancreas, liver and so forth,” Kamp said. “It’s pretty powerful technology to have this access to human cells.”
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The SCRMC has played a large role in establishing the university’s role at the forefront of the field. More than 600 scientists and students in almost 100 SCMRC labs around campus are working, teaching and studying in the field, according to a report from UW News.
The CRISPR-Cas9 is the latest generation of gene editing technology. This technology allows researchers to go into the IPS cells and edit the genome with great precision. Out of over a billion base pairs of deoxyribonucleic acid, the CRISPR-Cas9 can be programmed to sort through them and find the specific gene to edit. This allows researchers to investigate specific diseases and the genetic changes that are causing them.
Anita Bhattacharyya, Waisman Center investigator and assistant professor of cell and regenerative biology, co-directs the IPSC service with Su-Chun Zhang, professor of neuroscience and neurology at the Waisman Center.
“[The CRISPR-Cas9 gene editing approach is] easier to carry out than older methods of genetic engineering,” Bhattacharyya said. “It can target more places in the DNA of the genome so that scientists can edit parts of genes that they are interested in. Older genetic engineering strategies didn’t work well in human stem cells, while CRISPR-Cas9 does.”
Bhattacharyya has used the service for her own research into two genetic developmental disorders — Fragile X Syndrome and Down syndrome.
Down syndrome is caused by an extra chromosome, while Fragile X syndrome is due to a single gene mutation. Using IPS cells, scientist can study and define the mistakes in neurodevelopment that lead to these syndromes, and may be able to target treatments for these disorders.
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Kamp’s lab is currently using the gene editing service to study Long QT Syndrome, which is an inherited arrhythmia syndrome. His research focuses on stem cells and their applications to cardiovascular research and potentially cardioregenerative medicine.
While research does have long-term goals, such as therapy in the clinical field, investigators at this stage are focused on understanding the gene-editing process and testing it in cell culture models, not correcting genes in patients.
Regarding Chinese scientist He Jiankui’s recent claim that he had produced the world’s first gene-edited babies, the international scientific community was outraged that twin girls were born from embryos that were gene-edited for HIV resistance, as consensus in the scientific community holds that engineering human embryos for reproductive purposes should be prohibited until all scientific issues are resolved.
“This is a pretty powerful technology,” Kamp said. “With every big discovery like this, there is potential for advances and good to come, but there is also the chance of bad things to come. As a field, I think we are becoming more comfortable with the idea of treating diseases with some of these advanced technologies, but the concept of genetic engineering to make better, faster, stronger people brings about a lot of problems.”
The derivation of IPS cells has helped scientists avoid the ethical concerns of using human embryonic stem cells for research.
Though science hasn’t yet reached the ability to engineer humans, in the short term, the Human Stem Cell Gene Editing Service is enabling research projects by giving researchers the ability to use laboratory-made human stem cells to conduct research that is efficient in furthering knowledge on certain diseases.
“Hopefully, that will lead to new discoveries and advance therapies,” Kamp said. “We may be able to target diseases, or in the long-term, it may even be gene-editing in patients.”