Knock-Down Organoid Model for Target Profiling Analysis

By reducing the expression level of specific genes, it is possible to simulate the defects of these genes in normal physiological or disease states, thereby studying their effects on cellular processes, biological functions, or disease development. Knock-down studies contribute to revealing the roles of specific genes in signaling, metabolism, cell proliferation, differentiation, and biological development. They provide essential experimental evidence for understanding disease mechanisms, screening therapeutic targets, and drug development. To assist researchers in gaining a deeper understanding of gene function and disease mechanisms, as well as evaluating the effectiveness of potential therapeutic targets, Creative Biolabs offers knock-down organoid model construction and research services for target analysis.

What is the Knock-down Organoid Model?

Organoids are three-dimensional cellular aggregates that resemble organ structures and functions, derived from either stem cells or adult cells, and cultured in vitro. A knock-down organoid model is a method that utilizes gene editing techniques to downregulate specific genes within organoids.

Fig.1 Definition of organoid.¹

Advantages of Knock-down Organoid Model for Target Analysis

Accurate Disease State Simulation

Downregulating specific gene expression can simulate disease states or the impact of low gene expression on organ function, providing more accurate disease models.

Determination of Gene Function and Disease Mechanisms

Studying knock-down organoid models can reveal the role of target genes in the development of diseases.

Evaluation of Therapeutic Target Effects

Knock-down organoid models can be used to evaluate the effects of potential therapeutic targets, validating their role and efficacy in disease treatment.

Accelerated Drug Development

Knock-down organoid models can be utilized for drug target screening and efficacy assessment, expediting the drug development process, and providing important experimental data for the development and optimization of treatment strategies.

Knock-down Organoid Model Application Literature Sharing

• Title: DSCAM/PAK1 pathway suppression reverses neurogenesis deficits in iPSC-derived cerebral organoids from patients with Down syndrome (DS)
• Background: The authors discovered cortical developmental defects in cerebral organoids derived from induced pluripotent stem cells of patients with DS and identified an association with abnormal expression of the DSCAM signaling pathway.
• Model Used: Knock-down of DSCAM in DS cerebral organoids.
• Research Findings: Knock-down of the (DS cell adhesion molecule) DSCAM gene effectively rescued the reduced neural progenitor cell proliferation and cortical neurogenesis defects in DS cerebral organoids. Further pathway investigations revealed that the suppression of PAK1 (a downstream effector molecule of DSCAM) also rescued the cortical developmental defects in DS-derived organoids. These findings indicate a critical role of the DSCAM/PAK1 signaling pathway in regulating cortical development processes in DS, providing potential targets for the treatment of DS.

Fig.2 DSCAM-KD rescues impaired DSCAM/PAK1 signaling in DS-derived cortical cultures.2

Knock-down Organoid Model Construction Service for Target Analysis

Knock-down Organoid Model Construction Process at Creative Biolabs

• The customer specifies the target gene and organoid type for studies.
• Design and optimize the gene knock-down strategy.
• Culture organoids of the specified type.
• Construct the knock-down organoid model.
• Validate the knock-down efficiency of the target gene.
• Mechanistic studies /Drug screening.
• Deliver reports and interpret data at conferences.
• Question and answer session.

Creative Biolabs offers comprehensive services for the construction of knock-down organoid models corresponding to various organ types, providing clients with highly representative research tools. If you are currently seeking a model for target analysis, we encourage you to reach out to us for further information regarding knock-down organoid models. Moreover, in addition to the knock-down organoid model, we also provide services for the generation of the over-expression organoid model, knock-in organoid model, and knock-out organoid model.

Feel free to contact us to explore the full range of options available for your research needs.

Related Sections:

• Gene Editing Organoid Based Target Analysis and Validation Services
• Off-Target Screening Cell Microarray Assay