Precision guided therapy from a patient’s own cells

Dr. Wesley McKeithan, Stanford

Imagine having a tool you could use to quickly test lots of different drugs against a disease to see which one works best. That’s been a goal of stem cell researchers for many years but turning that idea into a reality hasn’t been easy. That may be about to change.

A team of CIRM-funded researchers at the Stanford Cardiovascular Institute and the Human BioMolecular Research Institute in San Diego found a way to use stem cells from patients with a life-threatening heart disease, to refine an existing therapy to make it more effective, with fewer side effects.

The disease in question is called long QT syndrome (LQTS). This is a heart rhythm condition that can cause fast, chaotic heartbeats. Some people with the condition have seizures. In some severe cases, particularly in younger people, LQTS can cause sudden death.

There are a number of medications that can help keep LQTS under control. One of these is mexiletine. It’s effective at stabilizing the heart’s rhythm, but it also comes with some side effects such as stomach pain, chest discomfort, drowsiness, headache, and nausea.

The team wanted to find a way to test different forms of that medication to see if they could find one that worked better and was safer to take. So they used induced pluripotent stem cells (iPSCs) from patients with LQTS to do just that.

iPSCs are cells that are made from human tissue – usually skin – that can then be turned into any other cell in the body. In this case, they took tissue from people with LQTS and then turned them into heart cells called cardiomyocytes, the kind affected by the disease. The beauty of this technique is that even though these cells came from another source, they now look and act like cardiomyocytes affected by LQTS.

Dr. Mark Mercola, Stanford

In a news release Stanford’s Dr. Mark Mercola, the senior author of the study, said using these kinds of cells gave them a powerful tool.

“Drugs for heart disease are typically developed using overly simplified models, like tumor cells engineered in a specific way to mimic a biochemical event. Consequently, drugs like this one, mexiletine, have undesirable properties of concern in treating patients. Here, we used cells from a patient to generate that person’s heart muscle cells in a dish so we could visualize both the good and bad effects of the drug.”

The researchers then used these man-made cardiomyocytes to test various drugs that were very similar in structure to mexiletine. They were looking for ones that could help stabilize the heart arrhythmia but didn’t produce the unpleasant side effects. And they found some promising candidates.

Study first author, Dr. Wesley McKeithan, says the bigger impact of the study is that they were able to show how this kind of cell from patients with a particular disease can be used to “guide drug development and identify better drug improvement and optimization in a large-scale manner.”

 “Our approach shows the feasibility of introducing human disease models early in the drug development pipeline and opens the door for precision drug design to improve therapies for patients.”

The study is published in the journal Cell Stem Cell.

Therapy developed with CIRM award used in new clinical trial for COVID-19

Dr. Joshua Rhein, Assistant Professor of Medicine in the University of Minnesota Medical School’s Division of Infectious Diseases and International Medicine
Image Credit: University of Minnesota

While doctors are still trying to better understand how to treat some of the most severe cases of COVID-19, researchers are looking at their current scientific “toolkit” to see if any potential therapies for other diseases could also help treat patients with COVID-19. One example of this is a treatment developed by Fate Therapeutics called FT516, which received support in its early stages from a Late Stage Preclinical grant awarded by CIRM.

FT516 uses induced pluripotent stem cells (iPSCs), which are a kind of stem cell made from reprogrammed skin or blood cells. These newly made stem cells have the potential to become any kind of cell in the body. For FT516, iPSCs are transformed into natural killer (NK) cells, which are a type of white blood cell that are a vital part of the immune system and play a role in fighting off viral infections.

Prior to the coronavirus pandemic, FT516 was used in a clinical trial to treat patients with acute myeloid leukemia (AML) and B-cell lymphoma, which are two different kinds of blood cancer.

Due to the natural ability of NK cells to fight off viruses, it is believed that FT516 may also help play a role in diminishing viral replication of the novel coronavirus in COVID-19 patients. In fact, Fate Therapeutics, in partnership with the University of Minnesota, has treated their first COVID-19 patient with FT516 in a new clinical trial.

In a news release, Dr. Joshua Rhein, Physician at the University of Minnesota running the trial site, elaborates on how FT516 could help COVID-19 patients.

“The medical research community has been mobilized to meet the unique challenges that COVID-19 presents. There are limited treatment options for COVID-19, and we have been inundated daily with reports of varying quality describing the potential of numerous therapies. We know that NK cells play an important role in responding to SARS-CoV-2, the virus responsible for COVID-19, and that these cells often become depleted in infected patients. Our intent is to replenish NK cells in order to restore a functional immune system and directly target the virus.”

In its own response to the coronavirus pandemic, CIRM has funded three clinical trials as part of $5 million in emergency funding for COVID-19 related projects. They include the following: a convalescent plasma study conducted by Dr. John Zaia at City of Hope, a treatment for acute respiratory distress syndrome (a serious and lethal consequence of COVID-19) conducted by Dr. Michael Matthay at UCSF, and a study that also uses NK cells to treat COVID-19 patients conducted by Dr. Xiaokui Zhang at Celularity Inc.  Visit our dashboard page to learn more about these clinical projects.

Stem cells used to promote quick and precise bone healing

A close-up view of the intricate microarchitecture of the pluripotent stem-cell-derived extracellular matrix. Image Credit: Carl Gregory/Texas A&M

Although some broken bones can be mended with the help of a cast, others require more complex treatments. Bone grafts, which can come from the patient’s own body or a donor, are used to transplant bone tissue to the injury site. However, these procedures can have setbacks such as increased recovery time and chronic pain. Each year approximately 600,000 people in the United States alone experience complications from bone healing.

Researchers at Texas A&M University found a way to use induced pluripotent stem cells (iPSCs), a type of stem cell that can turn into any cell type and can be derived from adults cells (e.g. skin cells), to create superior bone grafts. The team of researchers said these grafts could potentially be used to promote swift and precise bone healing, enabling patients to optimally benefit from surgical intervention.

The Texas A&M team used iPSCS to make mesenchymal stem cells (MSCs), which make the extracellular matrix needed for bone grafts. MSCs can be obtained from bone marrow, but they have a relatively shorter life span and are not as biologically active when compared to MSCs generated from iPSCs.

To test the effectiveness of their iPSC generated bone grafts, they implanted the extracellular matrix at a site of bone defects. After a few weeks, they found that their iPSC generated matrix was five to sixfold more effective than the best FDA-approved graft stimulator.

In a news release from Texas A&M, Dr. Roland Kaunas discusses the potential benefits of using iPSC generated bone grafts.

“Our material is very promising because the pluripotent stem cells can ideally generate many batches of the extracellular matrix from just a single donor which will greatly simplify the large-scale manufacturing of these bone grafts.”

Additionally, the Texas A&M team said this approach has the potential to be incorporated into numerous engineered implants, such as 3D-printed implants or metal screws, so that these parts integrate better with the surrounding bone.

The full results of this study were published in Nature Communications.

A brief video on bone grafts from Texas A&M is available below.

Super charging killer cells to fight leukemia

Colorized scanning electron micrograph of a natural killer cell.
Photo credit: National Institute of Allergy and Infectious Diseases

Racing car drivers are forever tinkering with their cars, trying to streamline them and soup up their engines because while fast is good, faster is better. Researchers do the same things with potential anti-cancer therapies, tinkering with them to make them safer and more readily available to patients while also boosting their ability to fight cancer.

That’s what researchers at the University of California San Diego (UCSD), in a CIRM-funded study, have done. They’ve taken immune system cells – with the already impressive name of ‘natural killer’ (NK) cells – and made them even deadlier to cancers.

These natural killer (NK) cells are considered one of our immune system’s frontline weapons against outside threats to our health, things like viruses and cancer. But sometimes the cancers manage to evade the NKs and spread throughout the body or, in the case of leukemia, throughout the blood.

Lots of researchers are looking at ways of taking a patient’s own NK cells and, in the lab boosting their ability to fight these cancers. However, using a patient’s own cells is both time consuming and very, very expensive.

Dan Kaufman MD

Dr. Dan Kaufman and his team at UCSD decided it would be better to try and develop an off-the-shelf approach, a therapy that could be mass produced from a single batch of NK cells and made available to anyone in need.

Using the iPSC method (which turns tissues like skin or blood into embryonic stem cell-like cells, capable of becoming any other cell in the body) they created a line of NK cells. Then they removed a gene called CISH which slows down the activities of cytokines, acting as a kind of brake or restraint on the immune system.

In a news release, Dr. Kaufman says removing CISH had a dramatic effect, boosting the power of the NK cells.

“We found that CISH-deleted iPSC-derived NK cells were able to effectively cure mice that harbor human leukemia cells, whereas mice treated with the unmodified NK cells died from the leukemia.”

Dr. Kaufman says the next step is to try and develop this approach for testing in people, to see if it can help people whose disease is not responding to conventional therapies.

“Importantly, iPSCs provide a stable platform for gene modification and since NK cells can be used as allogeneic cells (cells that come from donors) that do not need to be matched to individual patients, we can create a line of appropriately modified iPSC-derived NK cells suitable for treating hundreds or thousands of patients as a standardized, ‘off-the-shelf’ therapy.”

The study is published in the journal Cell Stem Cell.

You can bank on CIRM

Way back in 2013, the CIRM Board invested $32 million in a project to create an iPSC Bank. The goal was simple;  to collect tissue samples from people who have different diseases, turn those samples into high quality stem cell lines – the kind known as induced pluripotent stem cells (iPSC) – and create a facility where those lines can be stored and distributed to researchers who need them.

Fast forward almost seven years and that idea has now become the largest public iPSC bank in the world. The story of how that happened is the subject of a great article (by CIRM’s Dr. Stephen Lin) in the journal Science Direct.

Dr. Stephen Lin

In 2013 there was a real need for the bank. Scientists around the world were doing important research but many were creating the cells they used for that research in different ways. That made it hard to compare one study to another and come up with any kind of consistent finding. The iPSC Bank was designed to change that by creating one source for high quality cells, collected, processed and stored under a single, consistent method.

Tissue samples – either blood or skin – were collected from thousands of individuals around California. Each donor underwent a thorough consent process – including being shown a detailed brochure – to explain what iPS cells are and how the research would be done.

The diseases to be studied through this bank include:

  • Age-Related Macular Degeneration (AMD)
  • Alzheimer’s disease
  • Autism Spectrum Disorder (ASD)
  • Cardiomyopathies (heart conditions)
  • Cerebral Palsy
  • Diabetic Retinopathy
  • Epilepsy
  • Fatty Liver diseases
  • Hepatitis C (HCV)
  • Intellectual Disabilities
  • Primary Open Angle Glaucoma
  • Pulmonary Fibrosis

The samples were screened to make sure they were safe – for example the blood was tested for HBV and HIV – and then underwent rigorous quality control testing to make sure they met the highest standards.

Once approved the samples were then turned into iPSCs at a special facility at the Buck Institute in Novato and those lines were then made available to researchers around the world, both for-profit and non-profit entities.

Scientists are now able to use these cells for a wide variety of uses including disease modeling, drug discovery, drug development, and transplant studies in animal research models. It gives them a greater ability to study how a disease develops and progresses and to help discover and test new drugs or other therapies

The Bank, which is now run by FUJIFILM Cellular Dynamics, has become a powerful resource for studying genetic variation between individuals, helping scientists understand how disease and treatment vary in a diverse population. Both CIRM and Fuji Film are committed to making even more improvements and additions to the collection in the future to ensure this is a vital resource for researchers for years to come.

What to be thankful for this Thanksgiving: scientists hard at work

Biomedical technician Louis Pinedo feeds stem cells their special diet. Photo by Cedars-Sinai.

With Thanksgiving and Black Friday approaching in the next couple of days, we wanted to give thanks to all the scientists hard at work during this holiday weekend. Science does not sleep–the groundbreaking research and experiments that are being conducted do not take days off. There are tasks in the laboratory that need to be done daily otherwise months, even years, of important work can be lost in an instant.

Below is a story from Cedars-Sinai Medical Center that talks about one of these scientists, Louis Pinedo, that will be working during this holiday weekend.

Stem Cells Don’t Take the Day Off on Thanksgiving

Inside a Cedars-Sinai Laboratory, Where a Scientist Will be Busy Feeding Stem Cells During the Holiday

While most of us are stuffing ourselves with turkey and pumpkin pie at home on Thanksgiving Day, the staff at one Cedars-Sinai laboratory will be on the job, feeding stem cells.

“Stem cells do not observe national holidays,” says Loren Ornelas-Menendez, the manager of a lab that converts samples of adult skin and blood cells into stem cells—the amazing “factories” our bodies use to make our cells. These special cells help medical scientists learn how diseases develop and how they might be cured.

Stem cells are living creatures that must be hand-fed a special formula each day, monitored for defects and maintained at just the right temperature. And that means the cell lab is staffed every day, 52 weeks a year.

These cells are so needy that Ornelas-Menendez jokes: “Many people have dogs. We have stem cells.”

Millions of living stem cells are stored in the David and Janet Polak Foundation Stem Cell Core Laboratory at the Cedars-Sinai Board of Governors Regenerative Medicine Institute. Derived from hundreds of healthy donors and patients, they represent a catalogue of human ills, including diabetes, breast cancer, Alzheimer’s disease, Parkinson’s disease and Crohn’s disease.

Cedars-Sinai scientists rely on stem cells for many tasks: to make important discoveries about how our brains develop; to grow tiny versions of human tissues for research; and to create experimental treatments for blindness and neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS) that they are testing in clinical trials.

The lab’s main collection consists of induced pluripotent stem cells, or iPSCs, which mimic the all-powerful stem cells we all had as embryos. These ingenious cells, which Cedars-Sinai scientists genetically engineer from adult cells, can make any type of cell in the body—each one matching the DNA of the donor. Other types of stem cells in the lab make only one or two kinds of cells, such as brain or intestinal cells.

Handy and versatile as they are, stem cells are high-maintenance. A few types, such as those that make connective tissue cells for wound healing, can be fed as infrequently as every few days. But iPSCs require a daily meal to stay alive, plus daily culling to weed out cells that have started to turn into cells of the gut, brain, breast or other unwanted tissues.

So each day, lab staff suit up and remove trays of stem cells from incubators that are set at a cozy 98.6 degrees. Peering through microscopes, they carefully remove the “bad” cells to ensure the purity of the iPSCs they will provide to researchers at Cedars-Sinai and around the world.

While the cells get sorted, a special feeding formula is defrosting in a dozen bottles spread around a lab bench. The formula incudes sodium, glucose, vitamins and proteins. Using pipettes, employees squeeze the liquid into food wells inside little compartments that contain the iPSCs. Afterward, they return the cells to their incubators.

The lab’s 10 employees are on a rotating schedule that ensures the lab is staffed on weekends and holidays, not just weekdays. On Thanksgiving Day this year, biomedical technician Louis Pinedo expects to make a 100-mile round trip from his home in Oxnard, California, to spend several hours at work, filling nearly 600 feeding wells. On both Christmas and New Year’s Day, two employees are expected to staff the lab.

All this ceaseless effort helps make Cedars-Sinai one of the world’s top providers of iPSCs, renowned for consistency and quality. Among the lab’s many clients are major universities and the global Answer ALS project, which is using the cells in its search for a cure for this devastating disease.

That’s why the lab’s director, Dhruv Sareen, PhD, suggests that before you sit down to your Thanksgiving feast, why not lift a glass to these hard-working lab employees?

“One day the cells they tend could lead to treatments for diseases that have plagued humankind for centuries,” he says. “And that’s something to be truly thankful for.”

Blood-brain barrier chip created with stem cells expands potential for personalized medicine

An Organ-Chip used in the study to create a blood-brain barrier (BBB).

The brain is a complex part of the human body that allows for the formation of thoughts and consciousness. In many ways it is the essence of who we are as individuals. Because of its importance, our bodies have developed various layers of protection around this vital organ, one of which is called the blood-brain barrier (BBB).

The BBB is a thin border of various cell types around the brain that regulate what can enter the brain tissue through the bloodstream. Its primary purpose is to prevent toxins and other unwanted substances from entering the brain and damaging it. Unfortunately this barrier can also prevent helpful medications, designed to fix problems, from reaching the brain.

Several brain disorders, such as Amyotrophic Lateral Sclerosis (ALS – also known as Lou Gehrig’s disease), Parkinson’s Disease (PD), and Huntington’s Disease (HD) have been linked to defective BBBs that keep out critical biomolecules needed for healthy brain activity.

In a CIRM-funded study, a team at Cedars-Sinai Medical Center created a BBB through the use of stem cells and an Organ-Chip made from induced pluripotent stem cells (iPSCs). These are a specific type of stem cells that can turn into any type of cell in the body and can be generated from a person’s own cells. In this study, iPSCs were created from adult blood samples and used to make the neurons and other supporting cells that make up the BBB. These cells were then placed inside an Organ-Chip which recreates the environment that cells normally experience within the human body.

Inside the 3-D Organ-Chip, the cells were able to form a BBB that functions as it does in the body, with the ability to block entry of certain drugs. Most notably, when the BBB was generated from cell samples of patients with HD, the BBB malfunctioned in the same way that it does in patients with the disease.

These findings expand the potential for personalized medicine for various brain disorders linked to problems in the BBB. In a press release, Dr. Clive Svendsen, director of the Cedars-Sinai Board of Governors Regenerative Medicine Institute and senior author of the study, was quoted as saying,

“The study’s findings open a promising pathway for precision medicine. The possibility of using a patient-specific, multicellular model of a blood barrier on a chip represents a new standard for developing predictive, personalized medicine.”

The full results of the study were published in the scientific journal Cell Stem Cell.

First patient treated for colon cancer using reprogrammed adult cells

Dr. Sandip Patel (left) and Dr. Dan Kaufman (center) of UC San Diego School of Medicine enjoy a light-hearted moment before Derek Ruff (right) receives the first treatment for cancer using human-induced pluripotent stem cells (hiPSCs). Photo courtesy of UC San Diego Health.

For patients battling cancer for the first time, it can be quite a draining and grueling process. Many treatments are successful and patients go into remission. However, there are instances where the cancer returns in a much more aggressive form. Unfortunately, this was the case for Derek Ruff.

After being in remission for ten years, Derek’s cancer returned as Stage IV colon cancer, meaning that the cancer has spread from the colon to distant organs and tissues. According to statistics from Fight Colorectal Cancer, colorectal cancer is the 2nd leading cause of cancer death among men and women combined in the United States. 1 in 20 people will be diagnosed with colorectal cancer in their lifetime and it is estimated that there will be 140,250 new cases in 2019 alone. Fortunately, Derek was able to enroll in a groundbreaking clinical trial to combat his cancer.

In February 2019, as part of a clinical trial at the Moores Cancer Center at UC San Diego Health in collaboration with Fate Therapeutics, Derek became the first patient in the world to be treated for cancer with human-induced pluripotent stem cells (hiPSCs). hiPSCs are human adult cells, such as those found on the skin, that are reprogrammed into stem cells with the ability to turn into virtually any kind of cell. In this trial, hiPSCs were reprogrammed into natural killer (NK) cells, which are specialized immune cells that are very effective at killing cancer cells, and are aimed at treating Derek’s colon cancer.

A video clip from ABC 10 News San Diego features an interview with Derek and the groundbreaking work being done.

In a public release, Dr. Dan Kaufman, one of the lead investigators of this trial at UC San Diego School of Medicine, was quoted as saying,

“This is a landmark accomplishment for the field of stem cell-based medicine and cancer immunotherapy. This clinical trial represents the first use of cells produced from human induced pluripotent stem cells to better treat and fight cancer.”

In the past, CIRM has given Dr. Kaufman funding related to the development of NK cells. One was a $1.9 million grant for developing a different type of NK cell from hiPSCs, which could also potentially treat patients with lethal cancers. The second grant was a $4.7 million grant for developing NK cells from human embryonic stem cells (hESCs) to potentially treat patients with acute myelogenous leukemia (AML).

In the public release, Dr. Kaufman is also quoted as saying,

“This is a culmination of 15 years of work. My lab was the first to produce natural killer cells from human pluripotent stem cells. Together with Fate Therapeutics, we’ve been able to show in preclinical research that this new strategy to produce pluripotent stem cell-derived natural killer cells can effectively kill cancer cells in cell culture and in mouse models.”

A new stem cell derived tool for studying brain diseases

Sergiu Pasca’s three-dimensional culture makes it possible to watch how three different brain-cell types – oligodendrocytes (green), neurons (magenta) and astrocytes (blue) – interact in a dish as they do in a developing human  brain.
Courtesy of the Pasca lab

Neurological diseases are among the most daunting diagnoses for a patient to receive, because they impact how the individual interacts with their surroundings. Central to our ability to provide better treatment options for these patients, is scientists’ capability to understand the biological factors that influence disease development and progression. Researchers at the Stanford University School of Medicine have made an important step in providing neuroscientists a better tool to understand the brain.

While animal models are excellent systems to study the intricacies of different diseases, the ability to translate any findings to humans is relatively limited. The next best option is to study human stem cell derived tissues in the laboratory. The problem with the currently available laboratory-derived systems for studying the brain, however, is the limited longevity and diversity of neuronal cell types. Dr. Sergiu Pasca’s team was able to overcome these hurdles, as detailed in their study, published in the journal Nature Neuroscience.

A new approach

Specifically, Dr. Pasca’s group developed a method to differentiate or transform skin derived human induced pluripotent stem cells (iPSCs – which are capable of becoming any cell type) into brain-like structures that mimic how oligodendrocytes mature during brain development. Oligodendrocytes are most well known for their role in myelinating neurons, in effect creating a protective sheath around the cell to protect its ability to communicate with other brain cells. Studying oligodendrocytes in culture systems is challenging because they arise later in brain development, and it is difficult to generate and maintain them with other cell types found in the brain.

These scientists circumvented this problem by using a unique combination of growth factors and nutrients to culture the oligodendrocytes, and found that they behaved very similarly to oligodendrocytes isolated from humans. Most excitingly, they observed that the stem cell-derived oligodendrocytes were able to myelinate other neurons in the culture system. Therefore they were both physically and functionally similar to human oligodendrocytes.

Importantly, the scientists were also able to generate astrocytes alongside the oligodendrocytes. Astrocytes perform many important functions such as providing essential nutrients and directing the electrical signals that help cells in the brain communicate with each other. In a press release, Dr. Pasca explains the importance of generating multiple cell types in this in vitro system:

“We now have multiple cell types interacting in one single culture. This permits us to look close-up at how the main cellular players in the human brain are talking to each other.”

This in vitro or laboratory-developed system has the potential to help scientists better understand oligodendrocytes in the context of diseases such as multiple sclerosis and cerebral palsy, both of which stem from improper myelination of brain nerve cells.

This work was partially supported by a CIRM grant.

Midwest universities are making important tools to advance stem cell research

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iPSCs are not just pretty, they’re also pretty remarkable

Two Midwest universities are making headlines for their contributions to stem cell research. Both are developing important tools to advance this field of study, but in two unique ways.

Scientists at the University of Michigan (UM), have compiled an impressive repository of disease-specific stem cell lines. Cell lines are crucial tools for scientists to study the mechanics of different diseases and allows them to do so without animal models. While animal models have important benefits, such as the ability to study a disease within the context of a living mammal, insights gained from such models can be difficult to translate to humans and many diseases do not even have good models to use.

The stem cell lines generated at the Reproductive Sciences Program at UM, are thanks to numerous individuals who donated extra embryos they did not use for in vitro fertilization (IVF). Researchers at UM then screened these embryos for abnormalities associated with different types of disease and generated some 36 different stem cell lines. These have been donated to the National Institute of Health’s (NIH) Human Embryonic Stem Cell Registry, and include cell lines for diseases such as cystic fibrosis, Huntington’s Disease and hemophilia.

Using one such cell line, Dr. Peter Todd at UM, found that the genetic abnormality associated with Fragile X Syndrome, a genetic mutation that results in developmental delays and learning disabilities, can be corrected by using a novel biological tool. Because Fragile X Syndrome does not have a good animal model, this stem cell line was critical for improving our understanding of this disease.

In the next state over, at the University of Wisconsin-Madison (UWM), researchers are doing similar work but using induced pluripotent stem cells (iPSCs) for their work.

The Human Stem Cell Gene Editing Service has proved to be an important resource in expediting research projects across campus. They use CRISPR-Cas9 technology (an efficient method to mutate or edit the DNA of any organism), to generate human stem cell lines that contain disease specific mutations. Researchers use these cell lines to determine how the mutation affects cells and/or how to correct the cellular abnormality the mutation causes. Unlike the work at UM, these stem cell lines are derived from iPSCs  which can be generated from easy to obtain human samples, such as skin cells.

The gene editing services at UWM have already proved to be so popular in their short existence that they are considering expanding to be able to accommodate off-campus requests. This highlights the extent to which both CRISPR technology and stem cell research are being used to answer important scientific questions to advance our understanding of disease.

CIRM also created an iPSC bank that researchers can use to study different diseases. The  Induced Pluripotent Stem Cell (iPSC) Repository is  the largest repository of its kind in the world and is used by researchers across the globe.

The iPSC Repository was created by CIRM to house a collection of stem cells from thousands of individuals, some healthy, but some with diseases such as heart, lung or liver disease, or disorders such as autism. The goal is for scientists to use these cells to better understand diseases and develop and test new therapies to combat them. This provides an unprecedented opportunity to study the cell types from patients that are affected in disease, but for which cells cannot otherwise be easily obtained in large quantities.