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The new cell lines will advance research into common inherited heart diseases.
Seattle, June 4, 2024 Canada, July 18, 2020 /PRNewswire/ — The Allen Institute for Cell Science has launched a suite of tools to accelerate research into hypertrophic cardiomyopathy (HCM), the world’s most common inherited heart disease. A collection of six new cell lines, each harboring a different mutation associated with HCM..
HCM is primarily caused by mutations that thicken the heart muscle and, in rare cases, can lead to heart failure or cardiac arrest. The new cell line collection will help scientists investigate how specific mutations in myosin, the protein that helps the heart contract, affect heart function.
The institute also made available a collection of cell lines for skeletal muscle diseases linked to myosin mutations and for laminopathies, rare disorders caused by mutations in proteins that maintain the cell nucleus.
“Providing researchers with these powerful tools paves the way for the discovery of new treatments and insights into disease progression,” said Lu Gunawardane, PhD, executive director of the Allen Institute for Cell Science. “These cell lines are the perfect bridge between our expertise and the efforts of leading scientists tackling disease.”
Gene editing tools
The newest offering in the Allen Cell catalog is known as human induced pluripotent stem cells. These are skin cells that have been reprogrammed into a stem cell shape. Allen Institute scientists used CRISPR/Cas9 technology to introduce disease-associated mutations into these cells, as well as fluorescent tags that illuminate the structures that cause the heart to contract. Researchers can use these cells to generate cardiomyocytes, providing a dynamic model for investigating the complex biomechanics of heart disease.
The development of HCM tools provided an opportunity to try out new techniques for modifying single base pairs of DNA. Combining these gene editing methods allowed Allen Institute scientists to rapidly create a range of disease-specific cell lines. Brock Roberts“This is the beginning of a new era in human health, where scalpels and tools have gone to the molecular level,” said Allen Institute scientist Dr.
Collaborating in the lab for clinical advances
Today’s release of the HCM cell line: University of Washington; Stanford; and University of California, Santa BarbaraTheir goal was to investigate why mutations associated with HCM cause different symptoms in patients: some patients experience few or no symptoms, while others, even within the same family, suffer from severe disease. The team enlisted the help of the Allen Institute for Cell Science, which leveraged its decade of experience creating gene-edited stem cell lines to create a robust population of cells that could be used for this sensitive study.
“Working with the Allen Institute for Cell Science, we knew we would have access to high-quality stem cells.” Stanford University Daniel Bernstein“If we had done this on our own, it would have taken several years, been extremely expensive, and would have eaten up a large portion of the grant budget we had from the NIH,” MD said.
The cell line serves as a platform to study myosin mutations at the molecular, cellular and tissue levels, helping to comprehensively understand their impact on heart function. The researchers’ initial study confirmed that a specific rare mutation causes abnormal heart function at all biological levels, overturning previous skepticism about its clinical significance. “Some cardiologists were beginning to question whether this mutation was really the cause of disease, so being able to study this in a petri dish was very important,” said Bernstein, senior author of the study.
Cell lines also offer an opportunity to observe how a disease progresses over time, potentially revealing key stages where progression can be altered or halted by targeted intervention. “Most of the studies we do in human tissue are at late stages of the disease,” he says. Michael RegnierPhD, Professor of Bioengineering University of Washington “But what we really want to know is how the disease begins and how it progresses over time, and these cell lines make it possible to do that,” said a researcher involved in the study.
“A valuable resource”
Today’s announcement marks the first time that the Allen Cell Catalog has introduced disease-specific cell lines, and he hopes to see more in the future, he said. Jaclyn SmithSmith is a senior research scientist and has an MS degree. “As a research institute, we’ve been very focused on how normal cells function,” says Smith. “Now, by studying cells in disease states, we hope to gain a better understanding of these genetic conditions and develop new treatments.”
“Studying cardiomyocytes derived from cell lines from the Allen Institute for Cell Science allows us to understand how these molecular changes manifest at the sarcomere and cellular levels.” Kathleen RuppelM.D., Ph.D., Pediatric Cardiologist, Researcher Stanford University“These cell lines are an invaluable resource for myocardial research and will lead to important insights into hypertrophic cardiomyopathy.”
About the Allen Institute
The Allen Institute is an independent 501(c)(3) nonprofit research organization founded by the late philanthropist and visionary Paul G. Allen. The Allen Institute is committed to answering some of the biggest questions in bioscience and accelerating research worldwide. The Institute is a recognized leader in large-scale research committed to an open science model. Institutes and programs include the Allen Institute for Brain Science, established in 2003, the Allen Institute for Cell Science, established in 2014, the Allen Institute for Immunology, established in 2018, and the Allen Institute for Neurodynamics, established in 2021. In 2016, the Allen Institute expanded its reach with the creation of the Paul G. Allen Frontier Group, which unearths pioneers with new ideas to push the boundaries of knowledge and make the world better. For more information, visit alleninstitute.org.
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Source: Allen Institute
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