Advancing neuroscience with iPSC technology
Heine has always been interested in the development of the human brain. During her career, she has increasingly focused on early development and dedicated herself to working with stem cells. When the iPSC technology emerged, Heine launched a stem cell research project targeting children with vanishing white matter disease, one of the more common leukodystrophies. Her goal was to create early stem cells from patient-specific materials, which can be transplanted back to the patients, showcasing a promising application of iPSC in therapy.
In neuroscience, iPSCs are now widely used to create human brain and disease models that complement traditional animal models. “Results from animal studies often don’t translate well to human patients,” Heine explains. “Human iPSC-based models offer deeper insight into disease mechanisms and open the door to more effective therapies.”
The services of the iPSC Core Facility
The iPSC Core Facility provides a highly validated reprogramming service to convert somatic cells into top-quality iPSCs, comparable to embryonic stem cells. These cells can be differentiated into neurons, glial cells, or even 3D brain organoids, enabling researchers to study complex brain cell interactions.
Beyond the lab work, the Core Facility also provides support around the ethical aspects of iPSC research. As iPSCs are immortal and can be used indefinitely, it is critical to secure explicit, informed consent from donors. The facility assists researchers with documentation and ethical protocols, including consent forms developed in consultation with the METC. “This isn’t something to take lightly,” emphasizes the professor. “Donors must fully understand how their cells could be used – potentially forever.”
To manage these long-term commitments, the facility also operates the iPSC Biobank: a centralized repository for storing donor-derived cells and iPSC lines for current and future iPSC research.
As iPSCs can, in principle, divide endlessly, they offer a virtually unlimited supply of research material. This opens immense possibilities, but Heine highlights the importance of clear communication with donors and families. “The field is evolving quickly. People are willing to contribute—especially when it concerns brain disorders—but it’s our duty to ensure they’re truly informed.”
Personalized models with genetic precision
One of the most exciting applications of the iPSC technique is the ability to create personalized disease models that carry each donor’s unique genetic blueprint. “It is revolutionary”, says Heine. These models provide unmatched opportunities to explore genetic contributions to neurological and psychiatric conditions, from single-gene disorders to complex polygenic risk factors. Unlike animal models, iPSCs derived from patients naturally reflect the genetic variations in humans. This allows researchers to create more representative and individualized models, which can better predict treatment responses and accelerate the development of precision therapies. This translational approach is increasingly attracting interest from both academic and industry partners.
How iPSCs are made
Heine outlines the process: “Generating an iPSC line is a meticulous process. It begins with culturing donor cells and introducing reprogramming factors. Next, it's a matter of time to wait for the cells to change. We monitor them until colonies appear with the correct morphology of embryonic stem cells. The right cell colony is then isolated, grown, and characterized both at the molecular and cellular levels. This reprogramming process takes about three months. If gene editing is included, it adds another two to three months. All in all, generating a well-characterized iPSC line takes about six months, assuming everything runs smoothly. But once you have it, it is yours to use long-term in a wide variety of research applications.”
Partnering with the CRISPR Center
“The facility also collaborates closely with the CRISPR Center of Amsterdam UMC”, Heine continues enthusiastically. While patient-derived iPSCs carry the full genetic makeup of an individual, CRISPR gene editing enables researchers to introduce or correct a specific gene. This powerful combination helps isolate and study the effects of specific mutations, offering a precise and controlled way to explore disease mechanisms and validating therapeutic targets.
A collaborative hub within Amsterdam UMC
TheiPSC Core Facility is an integral part of Amsterdam UMC, providing centralized expertise, services, and infrastructure to support stem cell research across the institution and beyond. To further enhance interdisciplinary collaboration, the facility will soon transition to theRDC–Adore building, a cutting-edge research environment designed to bring together clinical and fundamental scientists under one roof.
In addition to providing high-quality iPSC lines and support with ethical protocols, the Core Facility also offershands-on training for researchers. “We now offer a two-day iPSC course,” Heine explains. “Participants learn the full workflow—from thawing and freezing to splitting and maintenance of iPSCs—under the guidance of our experienced technicians.” This practical training empowers researchers to work independently with iPSCs and promotes broader adoption of stem cell technologies across disciplines.
The road ahead
“The field is evolving rapidly,” says the professor. “It’s exciting to think where we’ll be in just a few years.” A major ambition of the iPSC Core Facility is to support the increasing shift toward personalized disease modeling. By generating patient-specific iPSC lines, researchers can build models that reflect individual genetic profiles, laying the foundation for more targeted and effective therapies.
While the facility focuses on generating and maintaining high-quality iPSC lines, across the field, researchers are increasingly using these lines to develop multi-organ platforms. This approach allows scientists to study how a person’s genetic makeup affects different tissues—such as the brain, heart, or gut—bringing new insight into the systemic nature of many diseases. Although tissue-specific differentiation is done by individual research labs, the Core Facility plays a vital role in enabling this work by providing the foundational iPSC lines.
At the same time, the field faces challenges around resource sharing and data standardization. “One of the bottlenecks in iPSC research is the lack of shared procedures,” Heine notes. To address this, the Core is working to establish standardized legal documentation, such as consent and material transfer agreements, and to improve data management and sharing frameworks. These efforts aim to make iPSC lines and associated data more reusable and interoperable, especially important in research on rare diseases, where every sample counts.
As data science continues to grow in influence, collecting data in a standardized and structured way becomes increasingly important. “We're not only providing cells—we’re helping to build a foundation for long-term, high-quality research,” Heine emphasizes. “It’s about creating a system where knowledge and resources can be reused, shared, and expanded across disciplines.”
In short
What is it?
Induced pluripotent stem cell (iPSC) technology enables the reprogramming of adult cells into a pluripotent state. These cells can be stored long-term and used to generate patient-specific models for biomedical research.
What can I do with it?
Create high-quality iPSC lines from donor material to develop human cell models or disease models. These models can be used to investigate biological mechanisms, study genetic disorders, or explore potential therapies.
Who can use this?
Internal and external interested parties.
Where is it?
Amsterdam UMC, location VUmc, Research and Diagnostic Center – Adore Building, Van der Boechorststraat 6b, 1081 BT Amsterdam.
How can I utilize the services of the iPSC Core Facility?
Visit the iPSC website to read about their services.
How to contact the iPSC Core Facility?
Get in contact with the iPSC team via iPSC@amsterdamumc.nl or fill in their contact form on the website.
Who is on the team?
iPSC Core Services is part of the Emma Center for Personalized Medicine.
Photo in header by Marieke de Lorijn.