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The molecular basis of Neurological therapies

Oggetto:

The molecular basis of Neurological therapies

Oggetto:

Academic year 2023/2024

Course ID
BIO0216A Pds 306-TB
Teacher
Tiziana Enrica Mongini (Lecturer)
Degree course
[0101M22] Molecular Biotechnology
Year
2nd year
Teaching period
First semester
Type
Related or integrative
Credits/Recognition
3
Course disciplinary sector (SSD)
MED/26 - neurology
Delivery
Formal authority
Language
English
Attendance
Obligatory
Type of examination
Written
Type of learning unit
corso
Oggetto:

Sommario del corso

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Course objectives

  • To learn the pathological and molecular basis of the wide spectrum of disorders affecting the central and peripheral nervous system;

  • to inform about the rules and options for research on rare diseases
  • to examine the status of art of consolidated therapeutical protocols;

  • to explore the new perspectives and the research challenges of upcoming new therapeutical approaches

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Results of learning outcomes

to be able to undestand the modern research approaches to therapies for several complex neurological disorders (i.e., neurodegeneration, genetic diseases, metabolica diseases) in the light of the new technologies 

to raise the attention and interest on rare diseases

to suggest new reasearch approaches for neurological orphan diseases 

 

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Program

Neurological disorders: classification and pathogenetic mechanisms

Rare diseases : definitions and regulatory rules

Old and new therapies in neurology : general overview on drugs and beyond: substrate modulation, immunomodulatiom, cell therapies, enzymatic therapies, gene therapies

State of the art for: genetic neuromuscular diseases (neuropathies, muscular dystrophies , spinal muscular atrophy), mitochondrial disorders, trinucleotide repeat expansion diseases,
neurometabolic diseases (leukodystrophies, lysosomal diseases), autoimmune diseases
(MS, myastenia gravis), neurodegenerative disorders (ALS, PD, dementias), ultra rare diseases

Future developments next to come or just on the horizon

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Course delivery

Frontal lessons supported by slides; clinical cases presentation; questions and answers 

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Learning assessment methods

Written examination, with ten open questions (three points each, maximum score 30); oral integration or alternative oral examination may be required

Suggested readings and bibliography

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A complete list of references including most recent reviews and original papers on the covered topics will be provided at the end of the course



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