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Gene therapy: nucleic acids to treat diseases, a new medicine emerging from multidisciplinary translational research - 20a/Série 3

Gene therapy: nucleic acids to treat diseases, a new medicine emerging from multidisciplinary translational research.

Faculté de gestion: Ecole doctorale (FBM-DOCT)

Responsable(s): Liliane Tenenbaum

Période de validité: 2015 ->

Horaires du cours (Apériodique)

Date Lieu Remarque Thématique Intervenant(s)
04.03.2020 de 14:00 à 15:45 Salle de séminaire 4 (BH08) Introduction on viral vectors for gene transfer Liliane Tenenbaum
06.03.2020 de 09:00 à 10:45 Salle de séminaire 4 (BH08) Historical overview of gene therapy. Successes and limitations. Liliane Tenenbaum
11.03.2020 de 14:00 à 15:45 Salle de séminaire 4 (BH08) Retroviral integration and consequences / Choice of articles for the final workshop. Angela Teresa Ciuffi, Liliane Tenenbaum
13.03.2020 de 09:00 à 10:45 Salle de séminaire 4 (BH08) Gene therapy for neurological diseases. Liliane Tenenbaum
18.03.2020 de 10:00 à 11:45 Salle de séminaire 4 (BH08) Gene repair technologies Nicole Déglon
25.03.2020 de 10:00 à 11:45 Salle de séminaire 4 (BH08) Gene Therapy for retinal diseases? Corinne Kostic Bensadoun
27.03.2020 de 09:00 à 11:45 Salle de séminaire 4 (BH08) Workshop / students presentations Liliane Tenenbaum

Tutorial (optionnel)

Langue(s) d'enseignement: anglais
Public: Oui
Crédits: 1.00


To give an overview of gene therapy paradigms and highlight how these are evolving with new developments in virology and molecular biology. To illustrate how fundamental discoveries are translated into new tools and new developments in gene therapy.
To stimulate the critical reading of timely reports in a fast moving area of research. To confront different opinions and approaches.


Gene therapy clinical trials with a focus on the main challenges that have been and those which remain to be solved.
Examples taken in different therapeutical strategies : i) overexpression of a gene to correct a recessive genetic defect; overexpression of a beneficial gene as a drug to augment a protective function; iii) silencing of a dominant genetic defect using RNA interference ; iv) genome engineering to correct a mutation in situ ; v) ex vivo gene therapy.


Travail personnel : Oui
Présentation : Oui
Test final : Non
Évaluation de la participation par le tuteur: Oui


1) Gruntman AM and Flotte TR (2018) The rapidly evolving state of gene therapy, FASEB J32:1733-1740.

2) Auricchio A et al., (2017) The Future Looks Brighter After 25 Years of Retinal Gene Therapy. Hum Gene Ther 28:982-987; Planul A and Dalkara D(2017) Vectors and Gene Delivery to the retina. Annu.Rev.Vis.Sci. 3:121-140.

3) Mendell, JR et al. Single-dose gene replacement therpy for spinal muscular atrophy. (2017) New Engl J Med 377(18):1713-1722.; Al-Zaidy SA et al. (2019) AVXS-101 for SMA1: Comparative Study with a prospective Natural History Cohort. J Neuromuscul Dis doi: 10.3233/JND-190403

4) Porter DL et al (2015). Chimeric antigen receptor T cells persist and induce sustained remissions in relapsed refractory chronic lymphocytic leukemia. Sci Transl. med 7(303):303ra139;

5) Hacein-Bey-Abina S. (2014). A modified γ-retrovirus vector for X-linked severe combined immunodeficiency; N Engl J Med 371(15):1407-17

6) VandenDriessche T and Chuah MK (2017) Hemophilia Gene Therapy: Ready for Prime Time? Hum Gene Ther. 28:1013-1023; Perrin GQ et al., 2019. Update on clinical gene therayp for hemophilia. Blood 133: 407-414.
7) Maedler, ML & Gersbach CA (2016) Genome-editing Technologies for Gene and Cell Therapy. Molecular Therapy 24, 430-446.

Exigences du cursus d'études

Notions de bases en virologie, en immunologie et en biologie moléculaire.

Conditions d'octroi

Evaluation positive de la participation active par le tuteur.

Conditions d'accès

Inscription: auprès de l'Ecole doctorale. Série 3.

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