se1072021 Engineered cells successfuly reat cardiovascular and pulmonary disease: Proof-o-conceptsludy shows possibilies of more cost. ScienceDaily Your source for the latest research news Science News from research organizations Engineered cells successfully treat cardiovascular and pulmonary disease Proof-of-concept study shows possibilities of more cost-effective cell therapy using ‘universal’ stem cells Date: July 6, 2021 Source: University of California - San Francisco Summary: Ina study in mice, scientists have shown that gene-edited cellular therapeutics can be used to successfully treat cardiovascular and pulmonary diseases, potentially paving the way for developing less expensive cellular therapies to treat diseases for which there are currently few viable options. she: f WD in G FULL STORY Scientists at UC San Francisco have shown that gene-edited cellular therapeutics can be used to successfully treat cardiovascular and pulmonary diseases, potentially paving the way for developing less expensive cellular therapies to treat diseases for which there are currently few viable options. The study, in mice, is the first in the emerging field of regenerative cell therapy to show that products from specially engineered induced pluripotent stom cells called "HIP" cells can successfully be employed to treat major diseases while evading the immune system. The findings subvert the immune response that is a major cause of transplant failure and poses a barrier to using engineered cells as therapy. “We showed that immune-engineered HIP cells reliably evade immune rejection in mice with different tissue types, a situation similar to the transplantation between unrelated human individuals. This immune evasion was maintained in diseased tissue and tissue with poor blood supply without the use of any immunosuppressive drugs.” said Tobias Deuse, MD, the Julien I.E, Hoffman, M.D. Endowed Chair in Cardiac Surgery and a first author of the study. Deuse’s research is an example of "living therapeutics," an emerging pillar of medicine in which treatments are broadly defined as living human and microbial cells that are selected, modified, or engineered to treat or cure disease. The study appears in Proceedings of the National Academy of Sciences. "Universal Stem Cells” Avoid Immune Detection hitps:slwwu.sciencedally.comireleases/2021107/210706115323,him 481610772021 Engineered calls successful ret cardiovascular and pulmonary disease: Prook-o-concept sud shows possibile of more cos. The prospects of generating specialized cells in a dish that can be transplanted into patients to treat various diseases are encouraging, the scientists report. However, the immune system would immediately recognize cells that were recovered from another individual and would reject the cells. Hence, some scientists believe that custom cell therapeutics need to be generated from scratch using a blood sample from every individual patient as starting material The research group at UCSF followed a different approach, using gene editing to create ‘universal stem cells’ (named HIP cells) that are not recognized by the immune system and can be used to make “universal cell therapeutics.” The team tested the abilty of these cells to treat three major diseases affecting different organ systems: Peripheral artery disease; chronic obstructive pulmonary disease from alphat-antitrypsin deficiency; and heart failure, increasingly a global epidemic with more than 5.7 milion patients in the United States alone and some 870,000 new cases annually The scientists transplanted specialized, immune-engineered HIP cells into mice with each of these conditions and were able to show that the cell therapeutics could alleviate peripheral artery disease in hindlimbs, prevent the development of lung disease in mice with alphat-antitrypsin deficiency, and alleviate heart failure in mice after myocardial infarction To enhance the translational aspect of this proof-of-concept study, the researchers assessed the treatment's efficacy using standard parameters for human clinical trials focusing on outcome and organ function The Promise of an Affordable Option Deuse, who is also surgical director of the Transcatheter Valve Program and directs Minimally Invasive Cardiac ‘Surgery, plans to explore the potential of these universal stems cells for treating other endocrine and cardiovascular conditions. He noted that, because of the novelty of the approach, a careful and measured introduction into clinical trials will be crucial. Once more is known about human safety, he said, it wll be easier to estimate when treatments using HIP cells might be approved and available for patients. One of the great benefits of this approach, said Deuse, is that the strategy of immune engineering comes with a reasonable price tag. It would make the manufacturing of universal, high-quality cell therapeutics more cost effective, could allow future treatment of larger patient populations, and facilitate access for patients from underserved communities. “In order for a therapeutic to have a broad impact, it needs to be affordable,” said Deuse. "That's why we focus ‘50 much on immune-engineering and the development of universal cells. Once the costs come down, the access for all patients in need increases.” ‘Sonja Schrepfer, MD, PhD, a senior author on the paper, is a UCSF professor who has joined Sana Biotechnology Inc. for the translation of the HIP technology into human therapeutics. She is a pioneer of immune engineering and co-inventor of the HIP cells The other co-senior author of the paper is Lewis L. Lanier, PhD, a UCSF professor of Microbiology and Immunology, and co-leader of the Cancer Immunology & Immunotherapy Program at the UCSF Helen Diller Family Comprehensive Cancer Center. Story Source: Materials provided by University of California - San Francisco. Note: Content may be edited for style and length. Journal Reference: 1, Tobias Deuse, Grigol Tediashvili, Xiaomeng Hu, Alessia Gravina, Annika Tamenang, Dong Wang, Andrew Connolly, Christian Mueller, Befiat Mallavia, Mark R. Looney, Malik Alawi, Lewis L. Lanier, Sonja Schrepfer. hitps:slwwu.sciencedally.comireleases/2021107/210706115323,him 215se1072021 Enginesred cells successfully reat cardiovascular ané pulmonary disease: Proot- concept study shows possibltes of more cost- Hypoimmune induced pluripotent stem cell-derived cell therapeutics treat cardiovascular and pulmonary diseases in immunocompetent allogeneic mice. Proceedings of the National Academy of Sciences, 2021; 118 (28): €2022091118 DOL: 10.1073/pnas.2022091118 This P: Cite This Page: MLA APA Chicago University of California - San Francisco. "Engineered cells successfully treat cardiovascular and pulmonary disease: Proof-of-concept study shows possibilities of more cost-effective cell therapy using ‘universal stem cells.” ScienceDaily, ScienceDaily, 6 July 2021. . RELATED STORIES Cone Snail Venom Shows Potential for Treating Severe Malaria Feb. 18, 2021 — Using venom from a cone snail, a new study suggests these conotoxins may potentially treat malaria. The study provides important leads toward the development of new and cost-effective anti-adhesion Researchers Ready B Cells for Novel Cell Therapy May 2, 2019 — Scientists are paving the way to use gene-edited B cells -- a type of white blood cell in the immune system ~ to treat a wide range of potential diseases that affect children, including hemophilia New Platform Poised to Be Next Generation of Genetic Medicines July 16, 2018 — Scientists have discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the Gene Therapy Via Skin Could Treat Many Diseases, Even Obesity ‘Aug. 3, 2017 — Scientist have now overcome challenges that have limited the use of gene therapy. 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