Gene editing with CRISPR-Cas9
Create precisely engineered cell models for disease modelling, target validation, and drug discovery
Gene editing with CRISPR-Cas9 allows researchers to introduce precise genetic changes into cells, making it possible to recreate or repair disease-causing mutations, investigate gene function, and evaluate new therapeutic approaches.
At Bioneer, we help biotech and pharmaceutical companies develop customized gene-edited cell lines and iPSC-based disease models that support drug discovery and translational research across a wide range of therapeutic areas and disease indications.
From project design and gene editing to clone selection, quality control, and cell banking, our scientists provide end-to-end support throughout the entire workflow.
Whether you are validating a novel drug target, investigating disease mechanisms, or developing advanced cellular models, we deliver reliable, well-characterized cell lines tailored to your research and screening efforts.
Applications of gene editing in drug discovery
– with CRISPR-Cas9
Gene editing is widely used throughout the drug discovery process to better understand disease biology and to develop more effective therapies.
Disease modelling
Introduce disease-associated mutations or reporter constructs into human cell models to replicate key aspects of disease biology and investigate disease mechanisms in a controlled setting.
Target validation
Correct, modify or remove specific genes to determine whether they play a causal role in disease and represent promising therapeutic targets.
Drug screening and efficacy testing
Evaluate potential drug candidates in genetically defined cellular models that better reflect patient biology and disease-relevant mechanisms.
Mechanistic studies
Investigate gene function, signaling pathways, and cellular processes through precise genetic modifications.
Isogenic controls
Generate matched cell lines that differ only by a specific genetic change, enabling more robust and reliable experimental comparisons.
Cell and gene therapy research
Support the development and evaluation of next-generation therapeutic approaches through customized engineered cell models.
Choose between a range of genetic modifications
Using the CRISPR-Cas9 system, we offer a range of genetic modifications:
- Single Base Pair Mutations: Introduction or removal of single bases, enabling the creation of perfect isogenic pairs (homozygous/heterozygous).
- Gene Knockouts: Precise removal of small gene segments to create out-of-frame deletions, leading to premature termination of protein expression.
- Sequence-Specific Deletions: Targeted removal or introduction of repeat structures or domains within endogenous genes.
- Reporter Cell Lines: Insertion of reporters (e.g., tags, GFP, RFP, resistance markers) as transcriptional or translational fusions.
- Inducible Gene Expression: Insertion of a gene-of-interest, controlled by an inducible promoter, into a safe-site locus such as AAVS1 or CLYBL.
- Inducible Gene Deletions: Using the CRE-Lox platform, gene deletions can be induced in differentiated cell lines when required.
A streamlined platform for clone screening and selection
We have established an optimized workflow for the efficient generation, identification, and expansion of gene-edited cell clones.
As illustrated below, the process begins with CRISPR-Cas9-mediated gene editing of hiPSCs, followed by single-cell sorting to isolate monoclonal populations.
Individual clones are expanded and screened using a combination of replica plating, DNA isolation, and genotyping to identify successfully modified cells.
Positive clones are then further expanded, subjected to rigorous quality control, and cryopreserved for long-term storage.
Our team of scientists has extensive experience in gene-editing workflows and clone characterization, ensuring the delivery of well-documented, high-quality monoclonal cell lines. With our streamlined screening platform, cell banking capabilities, and stringent quality control procedures, we provide reliable gene-modified clones for downstream research and development applications.
Other relevant services
- Optimized inducible expression system
- Cell bank maintenance
- QC processes for cell lines
FAQ: Understanding our gene editing services
Practical Questions
How long does a gene-editing project typically take?
Project timelines depend on the complexity of the genetic modification, the cell type, and the extent of clone screening and validation required. During the initial consultation, we provide a project-specific timeline based on your objectives.
What information do I need to provide before starting a project?
To design an efficient editing strategy, we typically discuss the target gene, desired modification (zygosity of the edit), cell type, intended downstream application, and any specific assay requirements. Our scientists can help refine the project scope during the planning phase.
How many edited clones do you usually deliver?
The number of clones varies depending on the project and customer requirements. We generally identify and characterize multiple positive clones, allowing selection of the most suitable candidates for downstream studies.
Can you help design the gene-editing strategy?
Yes. Our scientists support guide RNA design, donor template strategy, clone screening approaches, and experimental planning to maximize the likelihood of project success.
What documentation is included upon project completion?
Project deliverables typically include the edited cell line, clone-specific genotyping results, quality control data, and documentation relevant to the completed workflow.
Can projects be scaled as research needs evolve?
Yes. Many customers begin with a proof-of-concept project and later expand into additional edits, isogenic controls, disease models, or larger cell banking programs.
Can you generate homozygous and heterozygous mutations?
Yes. Depending on the editing strategy and biological requirements, we can screen for and identify clones carrying either heterozygous or homozygous modifications.
Can you edit multiple genes simultaneously?
In many cases, yes. Multiplex gene-editing strategies can be considered when the research question requires modification of multiple genetic targets.
How do you assess potential off-target effects?
Off-target assessment can be incorporated into project workflows depending on project requirements. The appropriate strategy is determined based on the edit, cell type, and intended downstream application.
Can you generate reporter cell lines for live-cell studies?
Yes. We can introduce fluorescent reporters, tags, or other genetic markers that enable visualization of gene expression, protein localization, or cellular activity.
Can gene-edited cell lines be differentiated into specific cell types?
Yes. Gene editing can be performed in hiPSCs before differentiation into disease-relevant cell types such as neurons, astrocytes, microglia, or other specialized cells.
Can you generate custom disease models outside CNS indications?
Yes. While we have extensive experience in CNS disease modelling, gene-editing services can be applied across a wide range of therapeutic areas and disease indications.
Can existing customer cell lines be used for gene editing?
In many cases, yes. We can evaluate customer-provided cell lines and determine whether they are suitable for the intended gene-editing workflow.
Do you offer cell banking and future clone recovery?
Yes. Edited clones can be cryopreserved and stored to ensure long-term availability and facilitate future project expansion.
When should we involve Bioneer in a gene-editing project?
We recommend engaging our scientists as early as possible. Early planning allows editing strategies, validation requirements, and downstream applications to be considered from the outset, helping reduce project risk and timelines.
Can Bioneer support both research and preclinical development programs?
Yes. Our services support discovery research, target validation, translational studies, and preclinical development programs requiring customized gene-edited cellular models.
Do you offer a free consultation before project initiation?
Yes. We are happy to discuss your project goals, evaluate technical feasibility, and suggest how we would approach your project before a project begins.
How is project communication managed?
Each project is supported by a dedicated project manager and experienced scientific team who provide regular updates and serve as your primary points of contact throughout the project.
Working with us
Partnering with us means working with a dedicated project manager and a team of experienced scientists who stay closely engaged throughout your project. We combine flexible workflows with a collaborative, human-centered approach to ensure your research moves forward efficiently and with clarity.
Established expertise
Bioneer has supported the life science community since 1982, building a strong foundation in science and technology and a solid track record in CNS research and disease modelling.
A human-centered way of working
We value close collaboration and long-term relationships, focusing on clear communication and trust at every stage of a project.
Our leadership
Bjørn Holst,
Dept. Head of Cellular Engineering and Disease Models
Kenneth Thirstrup,
R&D Manager, CNS Assays
Contact us for a free consultation
Interested in learning how we can support your project or pipeline?
We´re always happy to chat.
For further information please contact Business Development Manager Jacob Mathias Bech on jmb@bioneer.dk or +4523202576
For further information please contact Business Development Manager Jacob Mathias Bech on jmb@bioneer.dk or +4523202576
