Speakers invités

Frédéric Flamant 

Principal investigator. Groupe « Functional genomics of thyroid hormone receptors ». at Institut de Génomique Fonctionnelle de Lyon (IGFL) at Ecole Normale Supérieure de Lyon.

Since its fundation, the  group is focussed on the basic mechanisms of thyroid hormone action in neural cells. The main focus is on mouse genetics, for which the group established a leader position, generating and analyzing the phenotype of a large collection of transgenic models. In particular the development of the Cre/loxP technology for thyroid hormone receptor encoding genes allowed the group to fill the gap between cultured cell-based knowledge and animal studies. The group also pioneered genome scale studies of thyroid hormone receptors function, and currently applies this knowledge to the toxicology of thyroid hormone disruptors.

Carine Giovannangeli 

Carine Giovannangeli graduated in Physics from Ecole Normale Supérieure (Lyon) and got her PhD in Biophysics (University Paris VI) in 1992 on biophysical and biological characterization of unusual nucleic acid structures under the supervision of Claude Hélène. Afterwards she was hired by CNRS in 1992 and spent some years at the Pasteur Institute working on HIV and inhibitory strategies based on nucleic acids targeting of the viral genome. During the last few years at the Museum National d’Histoire Naturelle (Paris) she focused on optimizing genome editing methods using TALE proteins and CRISPR/Cas systems by characterizing the DNA repair mechanisms associated with nuclease driven genome modifications and providing resources in the field (TACGENE Facility).

Paula Rio

Paula Río is the Head of the Bone Marrow Aplasias Unit (Division of Hematopoietic Innovative Therapies, CIEMAT/CIBERER/IIS-FJD, UAM; Madrid, Spain). She has been working in Gene Therapy for Fanconi anemia for more than 20 years using lentiviral vectors and gene editing. Nowadays she is deeply involved as Associated investigator in two different clinical trials focused on the correction of HSCs from Fanconi anemia A patients (FANCOLEN, Eudra CT: 2011-006100-12 and FANCOLEN II EudraCT 2018-002502-31). She is also in charge of Production department of the GMP facility Clinistem for the development of gene therapy trials for rare diseases.

Since 2010 she has been also working in gene editing, first by targeting HSCs from FA patients by Homologous recombination and nowadays by Non-homologous End Joining using the CRISPR/Cas9 system and new editing tools.

She has published 52 articles (H index: 20) and received 6 International Awards including the Young Investigator Award by the European Society of Gene and Cell Therapy (ESGCT) in 2019 and

Paula has been for the last two years the Treasurer of the SETGYC and collaborates with the ESCGT in the Patients Information working group and as an abstract reviewer. Since 2020 she is a member of the Immuno-Gene Therapy Committee of the ISCT and participates actively in the Fanconi anemia Gene Therapy working group, organized by the Fanconi Anemia Research Fund. She has been recently nominated as a Board member of the Fanconi Anemia Research Fundation (FARF) and actively participates in the Fanconi anemia Gene Therapy working group. Since 2021 she is also member of the Gene Editing committee of the American Society of Gene and Cell Therapy (ASGCT) and member of the subcommittee on Emerging Gene and Cell Therapies of the American Society of Hematology (ASH).

David Bikard 

David is a young investigator at the Institut Pasteur in the department of Microbiology where he started his group in 2014. David graduated from AgroParisTech and obtained his PhD from Paris Diderot University for his work performed at the Institut Pasteur on the integron bacterial recombination system. He then joined the laboratory of Luciano Marraffini at the Rockefeller University as a postdoctoral fellow where he started to work on CRISPR systems. David is interested in applying engineering principles to better understand and fight pathogenic bacteria.

Cécile Martinat 

Over the last several years, C. Martinat has focused most of her efforts on the use of human pluripotent stem cells for pathological modeling of neuromuscular diseases. In particular, her group demonstrated that human embryonic stem cells carrying the causal mutation for Myotonic Dystrophy type 1 (DM1) can be used to identify new pathological mechanisms, and discover new therapeutic strategies. Her team has an extensive knowledge in the manipulation of human pluripotent stem cells and their differentiation into the main cell types involved in neuromusculair diseases. Her group has developed several powerful culture systems and image processing softwares for neuromuscular junction analysis.
Cécile Martinat is the head of INSERM UEVE UMR 861 and she is also the president of the French society on Stem Cell Research (FSSCR).

Mario Amendola 

Dr. Mario Amendola, Team Leader, Genethon (Evry, France) and CR INSERM. Dr. Amendola main interest is developing gene therapy and genome editing approaches for treating monogenic disorders. As a Ph.D. student he focused on the design, development and application of new LV to achieve efficient gene transfer and therapy in primary cells, including human HSC. During his post-doc he explored the field of chromatin dynamics and gene expression regulation and genome editing. Currently, as team leader in the UMR_S951, he is leveraging the gene transfer and genome editing tools to find novel genetic treatment for monogenic disorders.

Jussi Taipale 

Professor Jussi Taipale obtained his Ph.D. at the University of Helsinki in 1996 and continued at the University of Helsinki for his post doctorate before moving to Johns Hopkins University (Baltimore, MD, USA). Since 2003, he has headed an independent research laboratory focusing on systems biology of growth control and cancer. He has published more than 100 scientific articles of which 22 are in the most prestigious scientific journals (Nature, Science and Cell), won numerous awards and grants (e.g., Anders Jahre Prize for Young Researchers, EMBO Young Investigator, ERC Advanced Grant and Vetenskapsrådet Distinguished Professor Program) and is internationally recognized as a leader in the field of genomics and systems biology. In 2012, Professor Taipale was elected as Member of the Nobel Assembly at the Karolinska Institutet, which awards the Nobel Prize in Physiology or Medicine. In 2017, he took up the position of Herchel Smith Professor of Biochemistry at the University of Cambridge, UK and also maintains research groups at University of Helsinki, Finland and Karolinska Institutet, Sweden.

The Taipale group’s main expertise is in high-throughput biology – particularly in combining both experimental and computational approaches. The principal aim of the group is to understand two systems-level questions that are presently poorly understood: the mechanisms that control growth of tissues and organisms, and the rules that specify how DNA sequence determines when and where genes are expressed. The group has experience in high throughput screening using cDNA (Varjosalo et al. Cell 2008), RNA interference (Björklund et al. Nature 2006) and CRISPR (Haapaniemi et al., Nature Medicine 2018), and computational and experimental methods to identify causative regulatory variants and mutations (see Yin et al., Science 2017; Zhu et al., Nature 2018; Jolma et al., Cell 2013 and Nature 2015; Yan et al., Cell 2013 and Nature 2021). In addition, the Taipale group has extensive expertise on mouse models of gene and regulatory region function (see Dumont et al., Science 1998; Ma et al., Cell 2002; Hallikas et al. Cell 2006; Sur et al., Science 2012).

Marion Abecassis

Avocate en sciences de la vie admise au barreaux de Paris et New York, exerçant chez DLA Piper France LLP, Membre fondateur d'ARRIGE (Association for Responsible Research and Innovation in Genome Editing), Invitée permanente du Comité d’Ethique de l’INSERM.

Bernard Baertschi

Bernard Baertschi a enseigné la philosophie morale et la bioéthique à la Faculté de médecine et au Département de philosophie de l’Université de Genève jusqu’en 2014. En Suisse, il a été membre de la Commission fédérale d’éthique pour le génie génétique non humain (CENH) et, en France, il fait partie du Comité d’éthique de l'Inserm, ainsi que de celui de la Maison des Sciences de l’Homme. Il travaille actuellement sur les questions éthiques posées par les biotechnologies et les neurosciences, domaines dans lesquels il a notamment publié les ouvrages suivants: La neuroéthique (La Découverte, 2009), La vie artificielle (CENH, 2009), L'éthique à l'écoute des neurosciences (Les Belles-Lettres, 2013) et De l’humain augmenté au posthumain (Paris, Vrin, 2019).