Aim
CRISPR Based Gene Therapy covers how CRISPR systems are used to design gene therapies. Learn editing strategies, delivery systems, safety concepts, off-target control, and translation basics through case studies and a capstone therapy design plan (education/research use).
Program Objectives
- Gene Therapy Basics: targets, indications, in vivo vs ex vivo approaches.
- CRISPR Toolkit: Cas enzymes, gRNA design, repair pathways (NHEJ/HDR concepts).
- Therapy Strategies: knock-out, correction, base editing, prime editing (overview).
- Delivery: AAV, LNP, electroporation, viral/non-viral options (overview).
- Safety: off-target risk, genotoxicity concepts, immune response, biodistribution.
- Controls: specificity, validation assays, and quality checks (overview).
- Regulatory Basics: CMC, preclinical evidence, ethics, documentation.
- Capstone: design a CRISPR gene therapy plan for a disease target.
Program Structure
Module 1: Gene Therapy Foundations
- Gene therapy types: gene addition vs gene editing.
- Indications: monogenic disorders, oncology (overview).
- In vivo vs ex vivo workflows and decision criteria.
- Outcome measures and safety endpoints (intro).
Module 2: CRISPR Editing Mechanisms
- Cas enzymes, PAM, gRNA basics.
- DNA repair: NHEJ vs HDR concepts.
- Editing outcomes: indels, corrections, insertions (overview).
- Common failure modes and troubleshooting concepts.
Module 3: Advanced Editing Strategies (Overview)
- Base editing: point mutation correction concept.
- Prime editing: programmable edits concept.
- CRISPRi/CRISPRa for gene regulation (intro).
- Multiplex editing concepts and risks.
Module 4: gRNA Design and Off-Target Control
- gRNA design workflow: target selection and constraints.
- Off-target prediction concepts and scoring (overview).
- Reducing off-targets: enzyme choice, gRNA tuning, delivery control.
- Validation assays overview: sequencing-based checks.
Module 5: Delivery Systems for CRISPR Therapies
- Viral vectors: AAV and lentiviral concepts.
- Non-viral delivery: LNPs, polymers, electroporation concepts.
- Tissue targeting and biodistribution basics.
- Manufacturing constraints: size limits, stability, scalability (overview).
Module 6: Preclinical Evaluation (Safety and Efficacy)
- Efficacy: editing rate, expression rescue, functional assays (overview).
- Safety: off-targets, on-target rearrangements (concepts).
- Immunogenicity and inflammation risk concepts.
- Animal model selection and translational limits (overview).
Module 7: CMC, Quality, and Regulatory Basics
- Product definition: vector, payload, dose, route.
- QC concepts: purity, potency, identity, sterility (overview).
- Documentation: batch records, release criteria, traceability.
- Ethics: consent, long-term follow-up, risk communication.
Module 8: Clinical Translation and Case Studies
- Trial design basics: inclusion/exclusion, endpoints, monitoring.
- Risk-benefit thinking for high-impact indications.
- Patient safety monitoring and adverse event reporting (overview).
- Case study walkthroughs: strategy selection and evidence mapping.
Final Project
- Pick a target disease and gene.
- Deliverables: target rationale + edit strategy + delivery choice + safety plan + QC checklist.
- Submit: short therapy design dossier (education/research).
Participant Eligibility
- UG/PG students, PhD scholars in Biotechnology, Genetics, Molecular Biology, Pharmacy
- Researchers in gene editing and therapeutic development
- Basic genetics and molecular biology required
Program Outcomes
- Explain CRISPR strategies used in gene therapy.
- Select delivery methods and define key safety checks.
- Understand preclinical evidence and QC requirements.
- Create a CRISPR gene therapy design dossier.
Program Deliverables
- e-LMS Access: lessons, case studies, templates.
- Toolkit: gRNA design sheet, off-target checklist, safety/QC templates.
- Capstone Support: feedback on dossier.
- Assessment: certification after submission.
- e-Certification and e-Marksheet: digital credentials.
Future Career Prospects
- Gene Therapy Research Trainee
- CRISPR Scientist (Trainee)
- Biotech R&D Associate (Gene Editing)
- Translational Research Associate
Job Opportunities
- Biotech/Pharma: gene therapy and gene editing R&D teams.
- Academic Labs: functional genomics and therapeutic research projects.
- CROs: preclinical studies, sequencing validation, analytics support.
- Hospitals/Research Centers: translational research programs (non-clinical analytics).
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