Molecular Biology II
4 CFU
(Dorianna Sandonà)
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The course is planned for the Pharmaceutical Biotechnology Course students with a basic knowledge of molecular biology, especially regarding the structural organization of genes and the molecular mechanisms controlling transcription and translation. A good familiarity with the main recombinant DNA techniques is also required.
Objective:
This is an advanced course of molecular biology that aims to broaden the knowledge in the field of pharmaceutical biotechnology. In particular, the molecular mechanisms of several pathologies and the experiments planned to identify specific disease markers will be elucidated. By this approach, students will achieve the skill to design a methodological approach for the identification of markers for the diagnosis and prognosis as well as pharmacological targets of diseases. The molecular mechanism of action of some drugs able to cure defective genes will also be illustrated.
Content:
- Epigenetic and monoallelic gene expression
Epigenetic markers and "Histone code", genomic imprinting and developmental disorders, X chromosome inactivation, monoallelic gene expression, transposable elements, phenotypic consequences of transposable elements, epigenetic control of transposable elements.
- Post transcriptional gene regulation by small RNAs
The discovery of microRNA in C. elegans, expression, processing and mechanisms of action of microRNA, microRNA and organism development, microRNA and cancer, interfering RNAs, RNA interference as a tool to study genes, RNAi and therapeutic perspectives
- Molecular therapy of cancer
The molecular basis of cancer, activation of oncogenes, inactivation of tumor suppressors, chromosomal rearrangements and cancer, viruses and cancer, chemical carcinogenesis, cancer and epigenetic, new approaches for the molecular therapy of cancer, immunologic therapy of cancer, monoclonal antibodies, humanized antibodies, DNA vaccines.
- Treatments able to modify the RNA transcript or able to alter protein translation
Molecular basis of Duchenne muscular dystrophy and Becker muscular dystrophy, frame shift mutations nonsense mutations, exon skipping, therapeutic use of oligonucleotides for reading frame restore, oligonucleotides as drugs, the problem of oligonucleotides delivery, the nonsense mediated mRNA decay, stop codon read through, molecules able to induce stop codon read through.
Recommended texts:
the major part of the program is treated in: "Recombinant DNA" (3rd ed. JD Watson, AA Caudy, RM Myers and JA Witkowski; 2007) and "Fundamental of Molecular Biology" (1st ed LA Allison; 2007), for specific topics, recent experimental papers and reviews will be provided.

