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Advanced Therapies 2016
 
June 13 - 17
Luís Pereira de Almeida (luispa@cnc.uc.pt)

Lino da Silva Ferreira (Lino@biocant.pt)
PhD in Experimental Biology and Biomedicine
CNC - Center for Neuroscience and Cell Biology, University of Coimbra

Advanced Therapies 2016
June 13-17, 2016

Coordinators:

Luís Pereira de Almeida (luispa@cnc.uc.pt)
Lino da Silva Ferreira (Lino@biocant.pt)

Lecturers:
Lino Ferreira, CNC, UC
Ricardo Neves, CNC, UC
Filipe Pereira, CNC, UC
Hugo Fernandes, CNC, UC
Mário Grãos, Biocant
Sandra Pinto, CNC
Emanuel Quartin, CNC, UC
Adrian Jimenez, CNC, UC
Patrícia Pitrez, CNC, UC
Luís Estronca, CNC, UC,
Pedro Gouveia, CNC, UC
Henrique Almeida, CNC, UC
Helena Antunes, CNC, UC
Alessandra Zonari, CNC, UC
Catarina Almeida, CNC, UC
Akhilesh Rai, CNC, UC
Miguel Lino, CNC, UC
Susana Simões, CNC, UC
Artur Paiva, CHUC

João Nuno Moreira
Nuno Fonseca
Mariangela Natale

Ana Maria Cardoso
Olga Borges

Luis Pereira de Almeida
Clévio Nóbrega
Rui Nobre
Liliana Mendonça
Catarina Miranda
Ana Teresa Simões
Sónia Duarte
Carlos Matos
Joana Neves
Isabel Onofre
Sara Lopes
Janete Cunha-Santos
Vítor Carmona
Patrícia Albuquerque
Ana Cristina Ferreira

COURSE SYLLABUS

Monday, June 13th

9.00h-10.30h: Regenerative Medicine: from the bench to the bedside + stem cells and drug screening
Hugo Fernandes

11:00- 12:30h- Metabolic modulation can impact on stem cell physiology
Ricardo Neves

14.00h-15.30h- Biomaterials to modulate the activity of stem cells
Lino Ferreira

16:00h- 17:30h- Stem cells and mechanotransduction
Mário Grãos

Tuesday, June 14th


9.00h-10.30h: Programming and reprogramming cell fate + Programming definitive hematopoiesis
Filipe Pereira

11.00h-12.30h: High-throughput and high content screening
Sandra Pinto

14.00h-14.20h: Highlights of running projects I
Emanuel Quartin/ Adrian

14:20- 14:40h: Highlights of running projects II
Patricia Pitrez/Luis Estronca

14:40- 15:00h: Highlights of running projects III
Pedro Gouveia/Henrique Almeida

15:00- 15:20h: Highlights of running projects IV
Helena/Alessandra Zonari

15:20- 15:40h: Coffee break

15:40- 16:00h: Highlights of running projects V
Catarina Almeida/Akhilesh Rai

16:00- 16:20h: Highlights of running projects VI
Miguel Lino/Susana Simões

16:30- 17:30h- “Estudo da hematopoiese normal e displásica por citometria de Fluxo”
Paula Laranjeira/Artur Paiva (CHUCs)



Wednesday, June 15th


9.00h-10.30h: PLAYING AROUND WITH THE TUMOR MICROENVIRONMENT

    •    Vascular tumor targeting - João Nuno Moreira
    •    Targeted delivery of synergistic drug combinations: tackling cancer stem cells in human breast cancer - Nuno Fonseca
    •    The role of myofibroblasts in different microenvironment - Mariangela Natale

10:30- 11:00: Coffee break

11.00h-11.30h: LncRNAs as potential targets for cancer therapy: The case of lncRNA-MVIH metabolic regulation in GBM
Ana Maria Cardoso

11.30h-12.00h: In search of therapeutic vaccine to chronic hepatitis B
Olga Borges

12.00h- 12.45h: Exploring functional genomics for the development of RNA-based therapeutics
Miguel Mano

14.00h-14.40h: Molecular therapy of brain disorders
Luís Pereira de Almeida

14:40- 15:20: Understanding Molecular Mechanisms in Disease for the Development of Gene Targeting Therapeutic Approaches
Clévio Nóbrega

15:20- 15:50: Transplantation of Neural Stem Cells as a therapeutic strategy in Machado- Joseph disease
Liliana Mendonça

15:50- 16:00h: Coffee break

16:00- 16:30h: Mesenchymal stem cells in brain disorders
Catarina Miranda

16:30- 17:00h: Induced pluripotent stem cells to model neurodegeneration
Isabel Onofre

Thursday, June 16th


9.00h-9.30h: Inhibition of calpain-mediated proteolysis of mutant ataxin-3 as a therapeutic strategy in Machado-Joseph Disease
Ana Teresa Simões

9.30h-10.00h: NPY delivery as a strategy to alleviate MJD
Joana Neves

10.00h-10.30h: Pharmacological activation of autophagy
Ana Cristina Ferreira

10.30h-11.00h: Coffee Break

11.00h-11.30h: Caloric restriction alleviates neurodegeneration
Janete Cunha-Santos

11:30- 12:10: Silencing Machado-Joseph through the systemic route
Rui Nobre

14:00: 15:00 Exosomes as biomarker and gene carriers to the brain
Patrícia Albuquerque

15:00: 15:30 MicroRNA-mediated gene silencing in neurodegenerative disease
Vítor Carmona

15:30-16:00 Let-7 activates autophagy and alleviates MJD
Sónia Duarte

16:00- 16:15: Coffee break

16:15- 17:00h: Gene editing strategies in gene therapy
Sara Lopes and Carlos Matos

Friday, June 17th

9:00 – 12:30 Presentation of Papers/Projects



Background Reading:

Palsson, B.Ø. and Bhatia, S.N., Tissue Engineering, Pearson Prentice Hall Bioengineering, 2004;
Vunjak-Novakovic, G. and Freshney, R., Culture of Cells for Tissue Engineering, Wiley, 2006;
Atala, A., Lanza, R., et al, Principles of Regenerative Medicine, Academic Press, 2007;
Schaffer, D., Bronzino J.D., Peterson, D.R., Stem Cell Engineering, Principles and Practices, CRC Press, 2013;

Xu, J, Du, Y., Deng, H. Direct Lineage reprogramming: strategies, mechanisms, and applications. Cell Stem Cell 2015, 16, 119

Weber, J.  et al. 2014. From Pathways to Targets: Understanding the Mechanisms behind Polyglutamine Disease, BioMed Research International. Volume 2014, Article ID 701758.

Global Assessment of Stem Cell Engineering
(http://scec.gatech.edu/sites/default/files/StemCellEngineering-CompleteReport.pdf).

Regenerative Medicine 2006
(can be downloaded at http://stemcells.nih.gov/info/scireport/2006report.htm).

StemBook, http://www.stembook.org/, Harvard Stem Cell Institute





Assignments :

Students should present the assigned paper individually including a short proposal for a follow-up study. Presentations should not go over 20 min.
                       
João Amorim: Cao et al., Conversion of human fibroblasts into functional cardiomyocytes by small molecules. Science 10.1126/science.aaf1502 (2016).
                   
Luís Martins: Khacho et al. Mitochondrial Dynamics Impacts Stem Cell Identity and Fate Decisions by Regulating a Nuclear Transcriptional Program 2016, Cell Stem Cell 19, 1–16 August 4, 2016
               
M.ª Inês Martins: Banez-Coronel, M. et al. (2015). RAN Translation in Huntington Disease.  Neuron. 288(4):667-77. doi: 10.1016/j.neuron.2015.10.038.

Marcos Gomes: Mertens J, et al. Directly Reprogrammed Human Neurons Retain Aging-Associated Transcriptomic Signatures and Reveal Age-Related Nucleocytoplasmic Defects. Cell Stem Cell. 2015 Dec 3;17(6):705-18. doi: 10.1016/j.stem.2015.09.001.

Tiago Fonseca: Choudhury SR et al . Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector. Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28.
                                   
Ana Paula Rebelo: Paquet D, et al Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9. Nature. 2016 May 5;533(7601):125-9. doi: 10.1038/nature17664. Epub 2016 Apr 27.

Andreia Vilaça: Wahlquist et al. “Inhibition of miR-25 improves cardiac contractility in the failing heart”. Nature 2014, 508, 531.
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