CRISPR/Cas9 Point Mutation iPSC Service

Point Mutation Cell Lines mimic mutations seen in clinical specimens and are important in vitro disease models to understand role of genes in diseases (immuno-oncology, autoimmune diseases) as well as for drug discovery and immunotherapy development.

Leverage Applied StemCell unique expertise in CRISPR iPSC/ ES Cell Genome Editing Service to generate a point mutation iPSC line in your gene of interest:

• Send us your healthy/ patient iPSC or ESC lines; OR
• Choose from our well-characterized control “master” iPSC lines

Also available, downstream iPSC differentiation and characterization services.

• Optimized CRISPR protocols for high success rate
• Homozygous/ heterozygous point mutation clones
• Footprint-free, feeder-free transfection and iPSC culture protocols
• Isogenic control lines for reliable experiments

Workflow includes:

• Cell line evaluation
• gRNA design, CRISPR vector and donor DNA construction, and reagent validation
• Transfection of targeting vectors and optimization
• Screening for single cell clones and clone confirmation
• Cell expansion and cryopreservation

Deliverables:

• Two (2) clones for targeted point mutation, with two (2) vials of each clone at 1 x 10^6 cells/vial.
• Project Milestones Reports, as well as a Final Report containing details of targeting design, experimental, and genotyping results.

Timeline: 3-4 months

Applications:

• Physiologically relevant disease models for hard-to-model diseases (Ex. ALS, muscular dystrophy, Parkinson's disease, Alzheimer's)
• Differentiate to study mutations in different tissue lineages with an isogenic panel of cell line models
• Ideal for target drug discovery, drug and toxicity screening