ADVANCED THERAPIES MOD. 2
ADVANCED THERAPIES MOD. 2
| A.Y. | Credits |
|---|---|
| 2021/2022 | 2 |
| Lecturer | Office hours for students | |
|---|---|---|
| Luigia Rossi |
| Teaching in foreign languages |
|---|
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Course partially taught in a foreign language
This course is taught partially in Italian and partially in a foreign language. Study materials can be provided in the foreign language and the final exam can be taken in the foreign language. |
Assigned to the Degree Course
| Date | Time | Classroom / Location |
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| Date | Time | Classroom / Location |
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Learning Objectives
The course deals with an innovative therapeutic strategy based on the use of red blood cells as drug delivery systems: It consist in 2 parts: a) Red blood cells as drug delivey systems Part 1: From bench to beside; b) Red blood cells as drug delivey systems Part 2: PAL-loaded RBC for PKU treatment
Program
Red blood cells as drug delivery systems Part 1: From bench to beside
- Characteristics that make the red blood cell an ideal drug delivery system
- Temporary hypotonic lysis as the main method for loading molecules inside the erythrocytes
- Biomedical applications of erythrocytes as a drug delivery system
- Erythrocytes engineered for slow and sustained release of dexamethasone (Dexa)
- Automation of the loading process of a Dexa pro-drug in autologous blood
- Pilot clinical studies of Dex 21-P-loaded RBC in inflammatory diseases: COPD, CF, IBDs
- Phase II and phase III clinical studies of Dex 21-P-loaded RBC in patients with Ataxia telangiectasia
Red blood cells as drug delivery systems Part 2: PAL-loaded RBC for PKU treatment
- Genetic characteristics of phenylketonuria (PKU)
- Diagnosis of PKU
- Metabolism of phenylalanine (Phe) in healthy subjects and in subjects with PKU
- Pathological manifestations of PKU in the brain
- Classical therapeutic approaches for the treatment of PKU
- Enzyme therapy substituted and Palynziq: advantages and disadvantages
- Erythrocytes with the enzyme phenylalanine ammonium lyase (PAL) encapsulated as a therapeutic strategy capable of bypassing the adverse events of Palynziq
- Preclinical studies to evaluate the efficacy of PAL-loaded RBC in a mouse model of PKU
- Production, purification and characterization of the PAL wild type enzyme
- Further experimental therapies for the treatment of PKU
Teaching Material
The teaching material prepared by the lecturer in addition to recommended textbooks (such as for instance slides, lecture notes, exercises, bibliography) and communications from the lecturer specific to the course can be found inside the Moodle platform › blended.uniurb.it
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