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MOOC on gene and gene-based cell therapies for rare diseases

  • 3 hours ago
  • 2 min read

Foundation For Rare Diseases (Fondation Maladies Rares, France) organises an online MOOC on gene and gene-based cell therapies for rare diseases.

 

The course features contributions from leading U.S. and European researchers — including Alain Fischer, Heather Gray Edwards, Alessio Cantore, Zoltan Ivics, Sylvie Odent, Salima Hacein-Bey-Abina, Manuel Gonçalves, Marina Cavazzana, Allison Bradbury, Anne Galy, Chantal Pichon, Claire Booth, Jeffrey Medin, among others — as well as patients like Charlene Son Rigby, Victoria Gray, and Tesha Samuels, who shared powerful and valuable perspectives.

 

Key topics covered in the course 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

 

We are thrilled that the course has already reached 357 participants from 62 countries. We have also launched a facilitated learning window alongside the course rollout. During this period (March 16 – May 8) young post-docs in genetics and researchers (recruited mainly through ESHG-Y) are actively online, answering participants' questions and fostering dynamic engagement.

 

To broaden this impact further and reach more students, PhDs, and early-career researchers, we would be very grateful for your support through one or both of the following actions:

 

👉 New MOOC on FutureLearn: "From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases"

This course introduces the scientific and translational foundations of these advanced therapies, covering the genetics of rare disorders, key gene and cell engineering strategies, and the main steps in therapy development..

🧬Funded by ASGCT + Pfizer Medical Education Grant.


Opened: March 16 | Facilitated window (with experts): March 16 – May 8

Ideal for students, researchers, clinicians, and gene therapy enthusiasts. 👨‍🎓👩‍💻

 

Fondation Maladies Rares

96, rue Didot

75014 Paris (France)


Magda Granata, PhD

 


 


 
 
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