Doctoral Programme in Experimental Biology and Biomedicine
Course “Gene and Cell therapy of CNS: from microRNAs to IPS cells and gene repair”
January 7 to 11, 2013
This course will focus on the latest research in the field of gene and cell therapy, including viral and non-viral vectors, in situ and systemic delivery of nucleic acids, gene silencing, microRNAs and induced pluripotent stem cells. Some of the latest achievements in these domains will be presented by local and international experts in the scientific domain.
Monday (Jan 7) Gene Therapy
9:30-10:00- Presentation of the course, students and speakers
Luís Pereira de Almeida
10:00-12:00- Gene Therapy – Present and future perspectives
Luís Pereira de Almeida and Edgardo Rodriguez
12:00-14:30- Lunch
14:30-15:15- Gene Silencing
Edgardo Rodriguez
15:30-16:00- Silencing Machado-Joseph disease in the cerebellum
Clévio Nóbrega
16:00-16:30- Gene Delivery to the brain through non-invasive routes
Rui Nobre
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Tuesday (Jan 8) MicroRNAs
9:30-10:15 – Gene therapy of lysossomal diseases
Edgardo Rodriguez
10:30-11:00 – Non-viral-Gene therapy
Liliana Mendonça
11:30-12:30 – miRNAs as master regulators in cell biology and human disease
Francisco J. Enguita
12:30-14:30- Lunch
14:30 - 15:15 The dark (and funniest) side of the genome
Francisco J. Enguita
15:30 – 16:00 MicroRNAs and microglia
Ana Luísa Cardoso
16:00 – 16:30 MicroRNAs and glioblastoma
Pedro Costa
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Wednesday (Jan 9) Gene repair
9:30-10:30 - Editing the genome: from zinc fingers to tale nucleases
Neville Sanjana
10:30-11:00 – Break
11:00 -12:00 – Seminar: Edgardo Rodriguez
Splice isoform-specific suppression of the Cav2.1 variant underlying Spinocerebellar ataxia type 6.
12:00-14:30- Lunch
14:30-15:30 – Using optogenetics to dissect function and disease within the central nervous system
João Peça
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Thursday (Jan 10)
9:30-10:15 Reprogrammming fibroblasts into induced pluripotent stem cells and neurons
Isabel Onofre
10:30-11:00 – Stem Cells: the case of MSCs
Catarina Miranda
11:15-12:15 - Seminar: Using TALENs to model neurological disease in human pluripotent stem cells
Neville Sanjana
12:30-14:00- Lunch
14:00-15:00 – Engineering and scaling-up stem cell production
Margarida Serra
15:30-17:30- Paper presentations by students
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Friday (Jan 11)
9:30-12:30- Paper presentations by students
17:00-17:20- Multiple choice test
Invited lecturers:
Edgardo Rodriguez
Departments of Molecular Physiology and Biophysics, Internal Medicine and Neurology
University of Iowa,
Iowa City,
IA 52242 USA
Neville Sanjana
Broad Institute
7 Cambridge Center
Cambridge, MA 02142, USA
Francisco Enguita
Instituto de Medicina Molecular
Faculdade de Medicina,
Universidade de Lisboa,
Lisboa, Portugal
Margarida Serra
IBET- Instituto de Biologia Experimental e Tecnológica
Av. República, Qta. do Marquês (EAN)
2784-505 Oeiras- Portugal
CNC
Coordinator:
Luís Pereira de Almeida
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal
Lecturers:
Clévio Nóbrega
CNC
University of Coimbra
Coimbra, Portugal
Rui Nobre
CNC
University of Coimbra
Coimbra, Portugal
Ana Luísa Cardoso
CNC
University of Coimbra
Coimbra, Portugal
João Peça
CNC
University of Coimbra
Coimbra, Portugal
Pedro Costa
CNC & Faculty of Sciences and Technology
University of Coimbra
Coimbra, Portugal
Liliana Mendonça
CNC
University of Coimbra
Coimbra, Portugal
Isabel Onofre
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal
Papers for presentation and discussion:
10:30-12:30- Presentations (max. 20 min/group)
Group 1- Andreia Gomes, Maria do Céu Lourenço, Oksana Trotsenko
Paper 1
Group 2 - Dina Pereira, Mohamed Hussien, Cátia Santa,
Paper 2
Group 3 - Gladys Caldeira, RaviKumar Kapavarapu, Sandra Anjo
Paper 3
Group 4- Ivan Salazar, Sara Oliveira, Inês Santarino
Paper 4
Group 5- Jeannette Schmidt, Sofia Ferreira, Elda Bonifácio
Paper 5
Group 6- Lara Franco, Ana Cristina Ferreira, Maria Inês Sousa
Paper 6
Group 7- Susana Sampaio, Iryna Voytyuk, Helena Antunes
Paper 7
Group 8- Patrícia Morais, Eskedar Angamo, Josephine Blersch
Paper 8
Group 9- Vitor Carmona, Sara Silva, Geetha, Patricia Albuquerque
Paper 9
17:00-17:20- Multiple-choice examination
Students Assignments and Distribution
The assignments will include:
- Participation during the course
- Paper presentation & fictive projects (paper presentation, critical review of paper, proposal novelty, hypothesis-driven proposal, expected results)
- Multiple choice examination
Students are asked to consider an assigned paper as representing a pivotal work, briefly present it and prepare a brief project stating what experiments would be important to do next.
While briefly mentioning the paper findings, the students should concentrate on which directions the research would take. Apart from presenting data selected from the paper, presentation of the project should have the following points:
-Title
- State of the Art (include some data from the assigned paper)
-Objectives
- Tasks (maximum 3, including expected results and descriptions)
The fictive projects will be presented (20 min) and discussed (10 min) during Thursday afternoon and Friday morning (10:30-12:30)
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