Certificate of Achievement
has completed the following course:
From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases
This course explored gene and gene-modified cell therapies revolutionizing rare disease treatment. Learners examined the genetic foundations and therapeutic challenges of rare diseases affecting millions worldwide. Topics included viral vector technologies, gene editing approaches, mRNA therapeutics, and antisense oligonucleotides. The course covered clinical applications through real-world case studies, orphan drug designation frameworks, and implementation challenges, including patient access.
3 weeks, 6 hours per week
Daniel Scherman
Professor of Gene therapy, Director of the Foundation for Rare Diseases
Foundation for Rare Diseases
Transcript
Learning outcomes
- Describe the landscape of rare diseases, their genetic basis, and current therapeutic gaps
- Explain viral vector technologies and their mechanisms of gene delivery
- Compare gene editing approaches including CRISPR, base editing, and prime editing
- Identify mRNA therapeutics and antisense oligonucleotides as emerging treatment strategies
- Explore clinical case studies demonstrating successful gene therapy applications
- Evaluate orphan drug designation programs and regulatory frameworks
- Discuss implementation barriers and future directions in gene therapy for rare diseases
Syllabus
- Rare disease landscape: definitions, epidemiology, genetic origins, and therapeutic challenges
- Scientific and clinical rationale for gene and gene-modified cell therapies, including viral and non-viral vector-based gene delivery, gene editing technologies, mRNA-based therapies, antisense oligonucleotides (ASOs), and gene-modified cell therapies
- Clinical case studies showcasing successful applications of advanced therapies
- Key challenges in the development and implementation of gene-based therapies, including genotoxicity, immunotoxicity, and regulatory considerations
In association with
Issued on 10th July 2026
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