From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases
Discover how gene and gene-modified cell therapies transform rare disease treatment, from molecular mechanisms to clinical success
Duration
3 weeksWeekly study
6 hoursLanguages
English, Italiano, Español, Français, PortuguêsEnglish, Italiano, Español100% online
How it worksDigital upgrade
Free
From DNA to Cure: Gene and Gene-Modified Cell Therapies for Rare Diseases
This course has been certified by the CPD Certification Service as conforming to continuing professional development principles. Find out more.
Explore gene-based treatments for rare diseases
Explore gene and cell therapies transforming care for 300 million people with rare diseases worldwide.
On this three-week course from the Foundation for Rare Diseases you’ll study diverse gene therapy technologies, and clinical successes that have transformed lives.
Discover the science behind breakthrough therapies
95% of rare diseases lack treatments, which remain mostly symptomatic. In this course, you’ll explore the characteristics of rare diseases and understand why gene therapies offer an important opportunity to treat the disease directly at its root.
Master the technologies revolutionizing treatment
Dive into key technologies while being guided by international experts. You’ll examine: non-viral delivery systems (lipid nanoparticles, transposons), viral vectors (adenoviral, AAV, lentiviral, retroviral), gene editing (CRISPR-Cas9, base editing, prime editing), RNA therapeutics (mRNA, antisense oligonucleotides, siRNAs), and gene-modified hematopoietic stem cell therapy for blood disorders.
Understand safety and regulatory frameworks
Develop a strong understanding of critical safety considerations including genotoxicity and immunotoxicity. You’ll also learn how orphan drug designation programs incentivize rare disease drug development through market exclusivity and regulatory support.
Witness therapies changing lives
Gain a fundamental understanding of how revolutionary therapies move from concept to life-changing reality. You’ll expand your knowledge through clinical case studies in hemophilia, sickle cell disease, Fabry disease, and metabolic disorders, you’ll see how these revolutionary therapies move from concept to life-changing reality.
Syllabus
Week 1
Introduction to gene and gene-modified cell therapies for rare diseases
Welcome & Introduction
Welcome to the course! In this opening week, you'll discover why this topic matters, meet the contributors and start your learning journey with key glossary terms and an overview of the week's topics.
Introduction to rare diseases
In this activity, you will be introduced to the concept of rare diseases, and the critical need for the developement of new therapeutic approaches.
Introduction to cell and gene therapy
In this module, you will explore the fundamental principles of gene and gene-modified cell therapies and discover their life changing impact through a patient testimony.
Test your knowledge
To close the first week, you will get a brief recap and challenge yourself with a quiz to see what you have learned.
Week 2
Gene and gene-modified cell therapies approaches
Welcome & Introduction
Welcome to the second week! In this activity, you will discover this week's topics and learn essential scientific concepts.
Non-viral gene therapy: tools and approaches
In this activity, you will explore one of the major approaches in gene and cell therapies: non-viral vectors.
Viral vectors for gene therapy
How do we harness natural systems to deliver therapeutic genes? This activity introduces the principles and applications of viral vectors in gene and cell therapies.
Gene editing approaches
Discover how scientists target and modify specific DNA sequences in living cells. In this activity, you will learn more about genome editing.
Gene therapy with mRNA and synthetic oligonucleotides
How can RNA and synthetic oligonucleotides modulate gene expression? Learn about the mechanisms of action of these molecules and their therapeutic uses.
Gene-modified hematopoietic stem cell therapy
In this activity, you'll discover the gene-modified cell therapy approaches based on hematopoietic stem cells. How can we use hematopoietic stem cells to treat disease? Explore these modules to find out.
Test your knowledge
To close the second week, you will get a brief recap and challenge yourself with a quiz to see what you have learned.
Week 3
Real clinical cases and challenges
Welcome & Introduction
Welcome to the third and last week of the course! In this activity, you will discover this week's topics.
Challenges related to the development of advanced therapies
Gene and gene-modified cell therapies are at the center of next-generation treatments for patients with rare diseases. What challenges can they present? This activity will give you key insights into these obstacles.
Incentives and Strategies for the Development of Therapies for Rare Diseases
What strategies have been developed to support the development of gene and cell therapies and bring them to patients? How do these strategies address the specific challenges of rare diseases field?
Clinical examples
In this activity, you'll explore major real-life clinical examples and learn how a clinical trial can transform lives through a patient testimony.
Test your knowledge
To finish the third week and wrap up the course, you will get a final summary and test your knowledge with a final quiz.
Who is this accredited by?
The CPD Certification Service was established in 1996 and is the leading independent CPD accreditation institution operating across industry sectors to complement the CPD policies of professional and academic bodies.
When would you like to start?
Start straight away and join a global classroom of learners. If the course hasn’t started yet you’ll see the future date listed below.
Available now
Learning on this course
On every step of the course you can meet other learners, share your ideas and join in with active discussions in the comments.
What will you achieve?
By the end of the course, you‘ll be able to...
- 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
Who is the course for?
The course is ideal for university students and early-career researchers in the fields of biology, life sciences, and medicine. It is also open to anyone seeking to learn more about gene and cell therapies for rare diseases.
This course has been developed through funding from the ASGCT & Pfizer Medical Education Grant 2023.
Who will you learn with?
I am passionate about science, particularly gene therapy and other biotherapies. I am an emeritus professor and head of the research supporting Rare Diseases Foundation,
PhD in genetics and project manager at the Foundation For Rare Diseases.
Magda Granata, PhD in molecular biology, scientific project manager at Foundation For Rare Diseases. I coordinate the development of multiple MOOCs on FutureLearn, bridging science and education.
Who developed the course?
Foundation for Rare Diseases
The Foundation for Rare Diseases is a French non-profit foundation based in Paris, dedicated to advancing research and knowledge in the field of rare diseases. It offers educational programs and learning resources to promote understanding, innovation, and collaboration in this vital area worldwide.
What's included?
Foundation for Rare Diseases are offering everyone who joins this course a free digital upgrade, so that you can experience the full benefits of studying online for free. This means that you get:
Learning on FutureLearn
Your learning, your rules
- Courses are split into weeks, activities, and steps to help you keep track of your learning
- Learn through a mix of bite-sized videos, long- and short-form articles, audio, and practical activities
- Stay motivated by using the Progress page to keep track of your step completion and assessment scores
Join a global classroom
- Experience the power of social learning, and get inspired by an international network of learners
- Share ideas with your peers and course educators on every step of the course
- Join the conversation by reading, @ing, liking, bookmarking, and replying to comments from others
Map your progress
- As you work through the course, use notifications and the Progress page to guide your learning
- Whenever you’re ready, mark each step as complete, you’re in control
- Complete 90% of course steps and all of the assessments to earn your certificate
Want to know more about learning on FutureLearn? Using FutureLearn
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