Scientific Profiles

Genethon relies on the help of many experts.

For example :

Director of Research, Head of Inserm Unit 951
Anne Galy, Ph.D.

Anne Galy heads an INSERM Research Group at Genethon, as well as a hematopoietic gene therapy translational program. She is qualified in pharmacy and received her doctorate in Immunology from Université de Lyon (1989). She worked in the USA biotech sector in Palo Alto (California) before becoming a Professor of Immunology at the School of Medicine and at the Karmanos Cancer Institute at the Wayne State University in Detroit, Michigan. In 2001, she joined INSERM as a Research Director. She currently runs INSERM Unit 951 at Genethon, which has links with Université d’Evry Val d’Essonne and the Ecole Pratique des Hautes Etudes. This research laboratory develops innovative treatments for diseases of the immune system and blood, and is particularly interested in mechanisms for immunization by viral gene transfer. Anne Galy has published more than 90 articles in international scientific journals.

Researcher
Isabelle Richard, Ph.D.

Isabelle Richard joined Genethon in 1996 and heads a research group which is developing innovative muscular dystrophy treatments and investigating genetics and the physiopathological mechanisms of genetically-inherited diseases.
She is also in charge of a translational muscular gene therapy program.
She is Director of Research at the CNRS, and obtained her PhD in Human Genetics from Université de Paris 7 (1996).
She is taking part in a European project called NMD-chip and has had 110 articles published in international scientific journals.

Researcher
Anna Buj-Bello, Ph.D.

Ana Buj Bello heads a research team at Genethon working on neuromuscular diseases, as well as a translational program investigating gene therapy for the treatment of myotubular myopathy. She received her degree in Medicine and Surgery from the University of Lleida, Spain, and a PhD in Neurosciences from the University of St Andrews (Great Britain). She also obtained a diploma in Myology from the University of Pierre et Marie Curie, France. Having done a post-doctorate at the Institut de Génétique et Biologie Moléculaire et Cellulaire (IGBMC), Illkirch, where she worked for several years on the pathophysiology of myotubular myopathy, she joined the INSERM in 2004 and has worked at Genethon since 2009. Ana Buj Bello has published about 40 articles in international scientific journals and received the 2015 Outstanding New Investigator Award from the American Society of Gene & Cell Therapy (ASGCT).


Head of Applied Vector Studies
Otto-Wilhelm MERTEN, Ph.D.

Otto-Wilhelm Merten joined Genethon in 1997 and is currently in charge of the Applied Vector Studies group which helps innovate and optimize vector production. A Doctor of Biotechnology (Vienna, Austria), he has worked at the Institut Pasteur (Paris, France) as well as at the Sandoz Research Institute (Vienna, Austria). He has helped develop virus production processes, first for vaccination (influenza, poliomyelitis, rabies), and them for treatment applications. Otto-Wilhelm Merten has had 112 articles published in international scientifc journals and is Editor in Chief of Cytotechnology. He has also been a member of the ESACT (European Society for Animal Cell Technology) Executive Committee for 16 years (including four years as President from 2001 to 2005).

F MINGOZZI_CH_03_WEB Researcher
Federico MINGOZZI, Ph.D.

Federico Mingozzi heads a research group at Genethon which is working on the development of liver-targeted gene therapies for inherited diseases and on the characterization of immunity and tolerance in gene transfer. He is also Associate Professor at the University Pierre and Marie Curie in Paris.

He received his Biology degree and Ph.D. In Biochemistry and Molecular Biology from the University of Ferrara, Italy. He also obtained a Master in Business Administration from Drexel University, United States. He spent over ten years in the United States at the Children’s Hospital of Philadelphia and University of Pennsylvania where he was involved in the early development of gene therapies based on the adeno-associated virus (AAV) vector platform. In his research, he characterized the mechanisms of immune tolerance induction mediated by AAV vector liver gene transfer. More recently he focused on the clinical development of AAV vector-based gene therapies for inherited diseases and on the study of immune responses directed against AAV vectors in human trials. He contributed to several first-in-human AAV gene transfer clinical trials for indications including hemophilia B, Leber’s Congenital Amaurosis, and lipoprotein lipase deficiency. He contributed to about 80 publications, including original research articles, reviews and commentaries, and book chapters.