Summary ELA Info special n°110
Vera Gallo (Italy) : Gene therapy of hematopoietic stem cells with a lentiviral vector for metachromatic leukodystrophy, clinical results in 33 patients.
Raúl Estévez (Spain) : Reversibility in leukoencephalopathy megalencephalic: molecular and therapeutic mechanisms.
David A Wenger (United-States) : We are getting closer to better treatment of Krabbe disease.
Elise YAZBECK (France): Minoryx study in France.
Vivi Heine (The Netherlands) : Modeling of 4H syndrome using patient stem cells.
Françoise Piguet (France) : Genetic diseases – workshop Leukodystrophies undetermined.

Funding Opportunity Announcement, the topics:
– Clinical trial/ preparation for clinical trials in leukodystrophies, including development of long-term effects indicators, characterization of biomarkers, imaging protocols, pharmacodynamic/ pharmacokinetic studies.
– Pre-clinical studies specifically testing therapies (gene, cell, enzymologies or pharmacological therapies) in leukodystrophies.
– Development of animal or cellular models (for example iPSC derived from patients) relevant to human leukodystrophies.
– Studies of the mechanisms responsible for leukodystrophies, in order to identify new therapeutic approaches.