With over 95% of rare diseases still lacking approved treatments, there is a pressing need for specialized education in next-generation therapeutics. To address this gap, the Foundation for Rare Diseases (FFRD) is excited to announce the launch of their new Massive Open Online Course (MOOC) on FutureLearn: “From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases.”
Coordinated by FFRD and developed with support from the ASGCT & Pfizer Global Medical Education Grant, this course brings together internationally recognized experts to guide participants through the latest technologies reshaping the medical landscape.
Key topics include:
- Gene Editing: CRISPR-Cas9, base editing, and prime editing
- RNA Therapeutics: mRNA and Antisense Oligonucleotides (ASOs)
- Delivery Systems: Viral vectors and non-viral systems
- Real-World Impact: Clinical case studies in hemophilia and metabolic disorders, alongside moving patient testimonies
Enrollment is open now: https://www.futurelearn.com/courses/from-dna-to-cure-gene-and-gene-modified-cell-therapies-for-rare-diseases
Access to the content is free for the first 3 weeks after enrollment. A facilitated session runs from 16 March to 8 May. During this time, experts will be online to answer questions and support learners in real time.
This course is primarily designed for university students and early-career researchers in biology, life sciences, and medicine. It may also be of interest to healthcare professionals, policy makers, industry representatives, patient advocates, and anyone seeking to better understand the development of gene and gene-modified cell therapies for rare diseases. A basic background in biology is recommended.