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Gene and Cell therapy of CNS: from microRNAs to iPS cells and gene repair
 
January 7 - 11
Luís Almeida - luispa@ci.uc.pt, Clévio Nóbrega, Rui Nobre, Liliana Mendonça, Lígia Ferreira, Ana Luísa Cardoso, Catarina Miranda

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

__________________________________________________________________________

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

__________________________________________________________________________

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

__________________________________________________________________________

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

___________________________________________________________________________

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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