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Gene & Cell Therapy of CNS: from microRNAs to IPS Cells
 
January 30 - February 3
Luis Almeida
Doctoral Programme in Experimental Biology and Biomedicine
Course “Gene and Cell therapy of CNS: from microRNAs to IPS cells”
January 30 to February 3, 2012

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. The latest achievements in these domains will be presented by local and international experts in the scientific domain.

Monday (Jan 30)

10:00-10:30- Presentation of the course, students and speakers
Luís Pereira de Almeida

10:30-12:00- Gene Therapy – Present and future perspectives
Beverly Davidson

12:00-14:00- Lunch

15:00-16:00- Seminar - RNA therapeutics to the CNS
Beverly Davidson


Tuesday (Jan 31)


10:00-11:30 - Gene replacement therapy for inherited lysosomal disorders
Beverly Davidson

13:00-14:30- Lunch

14:30 - 15:15 miRNAs: from disease to therapeutic uses 
Francisco J. Enguita
15:15 – 15:30 Break
15:30 – 16:15 MicroRNA bioinformatics tools – fundaments and hands on
Francisco J. Enguita



Wednesday (Feb 1)

9:30-11:00- Different flavours of pluripotency, molecular mechanisms, and practical implications.
Niels Geijsen

11:00-11:15- Break

11:15-12:30- Engineering and scaling-up viral vector production
Paula Alves

12:30-14:00- Lunch

14:15-15:15- Engineering and scaling-up stem cell production
Paula Alves

15:15-15:30- Break

15:30-16:30 – Seminar: IPS cells: Programming and Reprogramming 
Niels Geijsen



Thursday (Feb 2)

9:30-10:00- Lentiviral vectors for gene therapy of the CNS
Luís Pereira de Almeida

10:00 - 10:30 – Silencing Machado-Joseph disease in the cerebellum
Clévio Nóbrega

10:30-10:45- Break

10:45-11:15 Gene Delivery to the brain through non-invasive routes
Rui Nobre

11:15-11:45  - Non-viral-Gene therapy
Liliana Mendonça

11:45-12:05 Gene transfer-mediated blockage of proteolysis
Ana Teresa Simões

12:05-12:25 Molecular and pharmacological blockage of adenosine receptors
Nélio Gonçalves

12:25-14:30- Lunch

14:30-14:50- Machado-Joseph disease ips cells
Isabel Onofre

14:50-15:00- Break

15:00-15:30- MicroRNAs and microglia

Ana Luísa Cardoso

15:30-15:50- MicroRNAs and glioblastoma

Pedro Costa

15:50-16:00- Break

16:00-17:00 – Editing the genome: from zinc fingers to tale nucleases (by videoconference)
Le Cong



Friday (Feb 3)

10:00-10:20- Multiple choice test

10:30-12:30- Paper presentations by students



Lecturers:

Beverly Davidson
Departments of Molecular Physiology and Biophysics, Internal Medicine and Neurology
University of Iowa,
Iowa City,
IA 52242 USA

Niels Geijsen
Utrecht University Veterinary School
& Hubrecht Institute; Uppsalalaan 8
3584 CT Utrecht; The Netherlands

Paula M. Alves
IBET- Instituto de Biologia Experimental e Tecnológica
Av. República, Qta. do Marquês (EAN)
2784-505 Oeiras- Portugal

Francisco Enguita
Instituto de Medicina Molecular
Faculdade de Medicina,
Universidade de Lisboa,
Lisboa, Portugal

Le Cong
Broad Institute
7 Cambridge Center
Cambridge, MA 02142, USA


CNC
Coordinator:
Luís Pereira de Almeida
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal

Lecturers:
Clévio Nóbrega
CNC
University of Coimbra
Coimbra, Portugal

Ana Luísa Cardoso
CNC
University of Coimbra
Coimbra, Portugal

Rui Nobre
CNC
University of Coimbra
Coimbra, Portugal

Liliana Mendonça
CNC
University of Coimbra
Coimbra, Portugal

Isabel Onofre
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal

Ana Teresa Simões
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal

Nélio Gonçalves
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal


Pedro Costa
CNC & Faculty of Sciences and Technology
University of Coimbra
Coimbra, Portugal



Papers for presentation and discussion:

10:00-10:20- Multiple-choice examination


10:20-10:30- Break


10:30-12:30- Presentations (max. 20 min/group)


Group 1- Ana Patricia Marques, Mariline Silva, António Carvalho Silva

Paper 1- Direct generation of functional dopaminergic neurons from mouse and human fibroblasts

Group 2- Joana Ribeiro Guedes, Catarina Almeida, Sara Lopes, Ana Isabel Santos

Paper 2- MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression



Group 3- Dominique Moreira Fernandes, Sofia Romano, Miguel Maria Lino

Paper 3- Conserved MicroRNA miR-8/miR-200 and Its Target USH/FOG2 Control Growth by Regulating PI3K

12:30-14:30- Lunch


14:30-15:30- Presentations 


Group 4- João Ribas Almeida, Tânia Perestrelo, Vanessa Machado

Paper 4- Adenosine deamination in human transcripts generates novel microRNA binding sites


Group 5- Marcelo Correia, André Filipe Soares, Daniel Santos

Paper 5- A TALE nuclease architecture for efficient genome editing



Group 6 - Catarina Almeida, Lazar Sopana, António Carvalho Silva
Paper 6 Microencapsulation Technology: A Powerful Tool for Integrating Expansion and Cryopreservation of Human Embryonic Stem Cells
Paper 6

Students Assignments and Distribution



The evaluation criteria will include:

-    Multiple choice examination 

-    Participation during the course

-    Paper presentation & fictive projects (paper presentation, critical review of paper, proposal novelty, hypothesis-driven proposal, expected results) 



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 Friday (14 October) morning (10:30-12:30) and afternoon (14:30-15:30).

Reference Papers

Alves_et_al_2010_HMG.pdf

Buecker&Geijsen_2010.pdf

Davidson_2011_Current prospects for RNA interference-based therapiesnrg2968-5.pdf

miRNA Regulatory Networks.pdf

Modular retroviral producer cell lines.pdf

Nascimento-Ferreira_et_al_2011_Brain.pdf

Nobre&Almeida_2011_CurrPharmDesig_proof.pdf

Noncoding RNA_human_disease_review M Esteller 2011.pdf

Pedroso_Lima_et_al_2005_CDT.pdf

Silva ac et al Adenovirus Vector Production and Purification.pdf

Tostoes_2012_Hepatology.pdf

Urnov et al 2010-Genome editing with engineered zinc finger nucleasesnrg2842-2.pdf


Other Papers

Buecker et al 2010

Hanna LIF-dependent hiPSCs supplement.pdf

Hanna LIF-dependent hiPSCs.pdf

Kobayashi and Nakauchi 2010

Supplemental Information Buecker et al.pdf

Wang and Liu 2011

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