CRISPR Course
Gain expertise in applying CRISPR to treat monogenic blood disorders. The course covers guide RNA design, off-target safety measures, HSC editing protocols, and validation assays, equipping you with hands-on skills to advance gene-editing research towards clinical applications in a safe and ethical manner.

from 4 to 360h flexible workload
valid certificate in your country
What will I learn?
This course provides a practical guide to designing and optimising precise single-base edits using CRISPR for monogenic blood disorders. Learners will master guide RNA selection, repair template design, base and prime editing techniques, ex vivo HSC workflows, delivery methods, molecular and functional assays, off-target analysis, genotoxicity checks, and key safety, ethical, and 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, base editing, or prime editing strategies.
- Optimise ex vivo HSC editing through RNP delivery, ideal culture conditions, and efficient scale-up processes.
- Validate gene edits comprehensively with NGS sequencing, allele-specific tests, and functional outcome assessments.
- Manage off-target effects and ethical concerns via safety assays, risk evaluations, and regulatory compliance.
Suggested summary
Before starting, you can change the chapters and 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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