Development of Individualized Therapies in Rare Genetic Disorders

Daten

Offizielle Daten in der Fachveröffentlichung für das folgende akademische Jahr: 2023-2024

Thematik

Rare diseases, while individually each disease is rare, collectively are common. More than 10,000 rare diseases affect nearly 400 million people worldwide. The personal and economic burdens of rare diseases are immense. Most of the rare diseases are caused by defective genes that impair quality of life and can lead to premature death. Gene therapy is a logical way to treat rare genetic diseases; cure a single gene defect by introducing a 'correct' gene. Today, there are only a handful therapies for rare diseases. As gene and other high-definition therapies are still under development, it is still unclear whether they will be successful in treating these diseases. In this course, we aim to address the potential and challenges of high-definition therapies for the treatment of rare genetic diseases.

Vorlesungen

• 1. Introduction to rare genetic disorders - Dr. Morava-Kozicz Éva
• 2. Introduction to rare genetic disorders - Dr. Morava-Kozicz Éva
• 3. Introduction to genetic therapies - Dr. Hadzsiev Kinga
• 4. Introduction to genetic therapies - Dr. Hadzsiev Kinga
• 5. The utility of induced pluripotent stem cell (iPSC) derived models in mitochondrial disease - Dr. Kozicz László Tamás
• 6. The utility of induced pluripotent stem cell (iPSC) derived models in mitochondrial disease - Dr. Kozicz László Tamás
• 7. Drug repurposing in congenital disorders of glycosylation - Dr. Morava-Kozicz Éva
• 8. Drug repurposing in congenital disorders of glycosylation - Dr. Morava-Kozicz Éva
• 9. Gene therapy in cardiomyopathy - Dr. Kozicz László Tamás
• 10. Gene therapy in cardiomyopathy - Dr. Kozicz László Tamás
• 11. Novel therapeutic approaches in rare disorders; liposomal targeting, mRNA treatment, ASO etc. - Dr. Morava-Kozicz Éva
• 12. Novel therapeutic approaches in rare disorders; liposomal targeting, mRNA treatment, ASO etc. - Dr. Morava-Kozicz Éva

Praktika

Seminare

Materialien zum Aneignen des Lehrstoffes

Obligatorische Literatur

Vom Institut veröffentlichter Lehrstoff

Az előadások anyagai.

Skript

Empfohlene Literatur

Voraussetzung zum Absolvieren des Semesters

Maximum of 15 % absence allowed

Semesteranforderungen

Beszámoló vagy vizsga

Möglichkeiten zur Nachholung der Fehlzeiten

Nincs lehetőség pótlásra.

Prüfungsfragen

Neptun Meet Street

Prüfer

Praktika, Seminarleiter/innen