![]() And exploiting these connections could help researchers establish new uses for iPSCs in human genetics, disease modeling, and drug discovery. This genotype–phenotype link may allow researchers to uncover once-hidden connections. It was very difficult to make genetic modifications.” He explains that new genome engineering tools (such as zinc finger nuclease, transcription activator-like effector nuclease, and CRISPR-Cas nuclease systems) have revolutionized researchers’ ability to introduce targeted genetic modifications that can reveal disease mechanisms and therapeutic targets.īy showing that a specific genotype (modification) is sufficient to make phenotypic change, Conklin notes, proof of the relationship can be provided. They could, for example, introduce disease-associated edits in iPSC lines or revert patient-derived iPSCs back to a wild-type genotype.Ī leader in the field, Bruce Conklin, MD, senior investigator at the Gladstone Institute of Data Science and Biotechnology at the University of California, San Francisco, tells GEN that “human iPSCs and human embryonic stem cells were very hard to do genetics with at first. According to Spraggon, there are clinical centers that have large repositories of iPSC lines that they are keen to edit. At present, however, momentum is building. Until recently, notes Lee Spraggon, PhD, head of cell engineering development at Synthego, there was an inertia to moving gene editing at scale into iPSCs. Together, they may bring about treatments far superior to those attainable with either discipline alone. Given the ability of patient-derived iPSCs to model diseases and the promise of gene editing to provide cures, some researchers are marrying the two disciplines. ![]() Stem cell research is hardly indifferent to genome editing, which is undergoing revolutionary change. Changing stem cells while maintaining their state In this article, due attention is given to some of the most significant stem cell advances: the application of CRISPR genome editing to iPSCs the development of stem cell therapeutics for diabetes and the creation of synthetic stem cell–derived embryos. These advances may not beguile a credulous public, but they are the most reliable indicators of progress. These cells, the company suggests, may benefit the animal’s health in the future.įar removed from stem cell services that offer only anecdotal evidence of efficacy, or no evidence at all, there are bona fide advances in stem cell research. The company obtains a dog’s stem cells from tissues collected during the animal’s routine spay or neuter procedure. For example, dog owners may be attracted to an animal biotech company called Gallant, which recently launched a stem cell banking service for dogs. Such products are suitable only for patients with diseases of the blood or bone marrow.ĭespite warnings from reputable scientists and regulatory authorities, people eager to benefit from regenerative medicine may seek care from stem cell clinics, if not for themselves, then for their beloved pets. The only stem cell–based products that have received FDA approval are those that consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood. Such therapies have been linked to serious complications. Besides objections to research involving human embryonic stem cells, there are concerns about unproven and unapproved stem cell–based therapies. ![]() The evolution of stem cell technology continues despite ongoing controversies. More recently, advances in biotechnology and genome editing have fostered a modern evolution of stem cell advances that shows no signs of slowing. Subsequent studies led to the isolation of human embryonic stem cells, which were then grown in the laboratory, as well as the development of reprogramming techniques and the generation of induced pluripotent stem cells (iPSCs). Accordingly, much has changed since the original revelation, the almost four-decade-old discovery of embryonic stem cells in mice. Just as one cannot step into the same river twice, one cannot find anything fixed in stem cell research, where one will find, instead, that revelations and still other revelations are ever flowing. ![]()
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