CRISPR Course
This course equips you with essential CRISPR techniques for treating monogenic blood disorders through precise single-base editing. You will master guide RNA design, editing strategies like HDR, base and prime editing, HSC workflows, validation assays, off-target control, and ethical compliance to advance gene therapy projects clinically.

flexible workload of 4 to 360h
valid certificate in your country
What will I learn?
Gain practical skills in designing and optimising precise single-base edits using CRISPR for monogenic blood disorders. Master guide RNA selection, repair template design, base and prime editing techniques, ex vivo HSC workflows, delivery methods, molecular assays, off-target analysis, genotoxicity checks, and key safety, ethical, regulatory aspects.
Elevify advantages
Develop skills
- Design effective CRISPR guides by selecting PAM sites, scoring on-target efficiency, and minimising bystander edits.
- Engineer accurate single-base corrections using HDR templates, base editors, or prime editing approaches.
- Optimise ex vivo HSC editing with RNP delivery, ideal culture conditions, and efficient scale-up processes.
- Validate gene edits thoroughly via NGS sequencing, allele-specific tests, and functional outcome assessments.
- Manage off-target effects and ethics through safety assays, risk evaluations, and regulatory compliance.
Suggested summary
Before starting, you can change the chapters and the workload. Choose which chapter to start with. Add or remove chapters. Increase or decrease the course workload.What our students say
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