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Gene Therapy and Nuclear Biology
 
January 11 - 15
Luis Almeida

luispa@ci.uc.pt
Course Schedule-“Gene Therapy and Nuclear Biology
January 11-15, 2010
 

Monday (Jan 11)
 
9:30-10:00- Presentation of the course, students and speakers
Luís Pereira de Almeida

10:00-11:00-  Gene Therapy- The A, B,C’s
Brian Kaspar
 
11:00-11:30- Break
 
11:30-12:30- Gene Therapy – Where do we stand?
Sérgio Simões
 
12:30-14:00- Lunch
 
14:00-15:00- Small regulating RNAs and silencing  
Ana Eulálio
 
15:00-15:45- Discussion and presentation of student’s assignments
 
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Tuesday (Jan 12)
 
9:30-10:30- Getting genes into the brain
Brian Kaspar
 
10:30-11:00- Break
 
11:00-12:00 – MicroRNAs mechanisms
Ana Eulálio
 
12:00-14:00- Lunch
 
14:00-15:00- Alternative splicing and disease
Margarida Gama Carvalho
 
15:00-15:15- Break
 
15:15-15:45- Nucleic acid-based strategies for the modulation of aberrant or alternative splicing in disease
Sara Trabulo
 
15:45-16:00- Discussion
 
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Wednesday (Jan 13)
 
9:30-10:30- Reprogramming of human neural stem cells into pluripotency.
Brian Kaspar
 
10:30-11:00- Break
 
11:00-12:00 – Lentiviral vectors for gene therapy of the CNS
Luís Pereira de Almeida
 
12:00-14:00- Lunch
 
14:00-14:30- Non-viral gene therapy to the CNS: DNA and siRNA delivery for the treatment of excitotoxic injury
Ana Luísa Cardoso
 
14:30-15:00- Development of gene delivery systems for application in antitumoral approaches involving a concerted action of gene therapy and chemotherapeutic agents
Henrique Faneca
 
15:00-15:15- Break
 
15:15-15:45- Gene therapy for Parkinson’s disease
Manuel Garrido
 
15:45-16:00- Gene therapy and food intake
Lígia Ferreira
 
16:00-16:15- Discussion
 

Thursday (Jan 14)
 
9:00-9:45- Use of Retroviral Vectors to Mark New Born Cells in the Brain
Brian Kaspar
 
9:45-10:00- Break
 
10:00-10:45- Gene therapy for pain
Isaura Tavares
 
10:45-11:00- Break
 
11:00-12:00- Engineering and scaling-up viral vector production I
Paula Alves
 
12:00-14:00- Lunch
 
14:00-15:00- Engineering and scaling-up viral vector production II
Paula Alves
 
15:00-15:15- Break
 
15:15-16:15 -Translation of gene therapies- The process from bench to bedside  
Brian Kaspar
 
16:15-16:45- Discussion
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Friday (Jan 15)
10:00-12:00- Paper presentations by students
 
16:00-17:00- Seminar: Gene Therapy Progress: Prospects for moving to Clinic in Neuromuscular Disorders
 Brian Kaspar
 
Lecturers:
 
Brian Kaspar
The Research Institute at Nationwide Children s Hospital
Center for Gene Therapy
700 Children s Drive, WA3010
Columbus, OH 43205, USA
 
Ana Eulálio
Max Planck Institute for Infection Biology
Charitéplatz 1
D-10117 Berlin
GERMANY
 
Margarida Gama Carvalho
Faculty of Medicine, University of Lisbon
and Center for Biodiversity, Functional and Integrative Genomics, Faculty of Sciences
University of Lisbon
 
Isaura Tavares
Faculty of Medicine, University of Porto
Alameda Hernâni Monteiro,
4200-319 Porto
 
Paula M. Alves
IBET- Instituto de Biologia Experimental e Tecnológica
Av. República, Qta. do Marquês (EAN)
2784-505 Oeiras- Portugal
 
 
CNC
Luís Pereira de Almeida
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal
 
Sérgio Simões
CNC &
Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal
 
Henrique Faneca
CNC& Department of Life Sciences
University of Coimbra
Coimbra, Portugal
 
Ana Luísa Cardoso
CNC
University of Coimbra
Coimbra, Portugal
 
Manuel Garrido
CNC
University of Coimbra
Coimbra, Portugal
 
Sara Trabulo
CNC & Department of Life Sciences
University of Coimbra
Coimbra, Portugal
 
Lígia Ferreira
CNC & Faculty of Pharmacy
University of Coimbra
Coimbra, Portugal
 
 
 
Papers for presentation and discussion:
 
Paper 1. Presentation by group 1 (Ana Carolina Moreira, Ana Luísa Pinho, Rui Costa). Questions by groups 5 and 6
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Duque S, Joussemet B, Riviere C, Marais T, Dubreil L, Douar AM, Fyfe J, Moullier P, Colle MA, Barkats M. Mol Ther. 2009 Jul;17(7):1187-96.
 
Presentation by group 2 (Ana Raquel Martins, Ângela Crespo, Ana Cardoso, Isabel Onofre). Questions by groups 3 and 4.
Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model. Kota J, Chivukula RR, O Donnell KA, Wentzel EA, Montgomery CL, Hwang HW, Chang TC, Vivekanandan P, Torbenson M, Clark KR, Mendell JR, Mendell JT. Cell. 2009 Jun 12;137(6):1005-17.
 
Presentation by group 3 (Carlos Rodrigues, Claudio Roque, Marta Passadouro). Questions by groups 1 and 2.
Sequence- and target-independent angiogenesis suppression by siRNA via TLR3. Kleinman ME, Yamada K, Takeda A, Chandrasekaran V, Nozaki M, Baffi JZ, Albuquerque RJ, Yamasaki S, Itaya M, Pan Y, Appukuttan B, Gibbs D, Yang Z, Karikó K, Ambati BK, Wilgus TA, DiPietro LA, Sakurai E, Zhang K, Smith JR, Taylor EW, Ambati J. Nature. 2008 Apr 3;452(7187):591-7.
 
Presentation by group 4 (Helena Leitão, Humberto Ferreira, Susana Pereira). Questions by groups 5 and 6.
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Brown BD, Gentner B, Cantore A, Colleoni S, Amendola M, Zingale A, Baccarini A, Lazzari G, Galli C, Naldini L. Nat Biotechnol. 2007 Dec;25(12):1457-67.
 
Presentation by group 5 (Marília Cordeiro, Miranda Mele, Sara Rito). Questions by groups 3 and 4.
Molecular therapy of obesity and diabetes by a physiological autoregulatory approach. Cao L, Lin EJ, Cahill MC, Wang C, Liu X, During MJ. Nat Med. 2009 Apr;15(4):447-54.
 
Presentation by group 6. Questions by groups 1 and 2 (Patrícia Monteiro, Paula Banca, Márcio Rodrigues). Yin H, Moulton HM, Seow Y, Boyd C, Boutilier J, Iverson P, Wood MJ. Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet. 2008 Dec 15;17(24):3909-18.
 
 
Reference Papers:
 
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Edelstein L, Abedi R, Wixon J. Gene therapy clinical trials worldwide to 2007- an update. J Gene Med. 2007;9(10):833-842.
 
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