title: Progress toward Treatment and Cure of Epidermolysis Bullosa: Summary of the DEBRA International Research Symposium EB2015.
abstract: Epidermolysis bullosa (EB), a group of complex heritable blistering diseases, is the topic of triennial research meetings organized by DEBRA International, Vienna, Austria, the network of national EB patient advocacy organizations. The DEBRA 2015 Research Conference, held in May 2015, brought together investigators and clinicians from around the world working at the forefront of EB research. Discussing the state-of-the-art approaches from a wide range of disciplines, there was a palpable excitement at this conference brought about by the optimism about applying new sequencing techniques, genome editing, protein replacement, autologous and allogeneic stem cell therapy, innovations in cancer biology, revertant mosaicism, and induced pluripotent stem cell techniques, all of which are aimed at developing new therapies for EB. Many in the field who have participated in EB research for many years were especially enthusiastic and felt that, possibly for the first time, the field seems uniquely poised to bring these new tools to effectively tackle EB. Multiple complementary approaches are currently in motion toward improved quality of life and eventually a cure for patients suffering from EB, a currently intractable disease.
authors.affiliation: Department of Dermatology and Cutaneous Biology, Thomas Jefferson University, Philadelphia, Pennsylvania, USA. Electronic address: Jouni.Uitto@jefferson.edu.
authors.forename: Jouni
authors.initials: J
authors.lastname: Uitto
authors.affiliation: Department of Dermatology, Medical Center, University of Freiburg, Freiburg, Germany.
authors.forename: Leena
authors.initials: L
authors.lastname: Bruckner-Tuderman
authors.affiliation: Department of Dermatology, Columbia University, New York, New York, USA.
authors.forename: Angela M
authors.initials: AM
authors.lastname: Christiano
authors.affiliation: Genetic Skin Disease Group, St. John's Institute of Dermatology, King's College London (Guy's Campus), London, UK.
authors.forename: John A
authors.initials: JA
authors.lastname: McGrath
authors.affiliation: Department of Dermatology, Medical Center, University of Freiburg, Freiburg, Germany.
authors.forename: Cristina
authors.initials: C
authors.lastname: Has
authors.affiliation: Department of Dermatology and Cutaneous Biology, Thomas Jefferson University, Philadelphia, Pennsylvania, USA.
authors.forename: Andrew P
authors.initials: AP
authors.lastname: South
authors.affiliation: DEBRA of America, New York, New York, USA.
authors.forename: Brett
authors.initials: B
authors.lastname: Kopelan
authors.affiliation: DEBRA International, Vienna, Austria.
authors.forename: E Clare
authors.initials: EC
authors.lastname: Robinson
mesh_terms: D000818:Animals; D001317:Austria; D016026:Bone Marrow Transplantation / Q000379:methods; D003226:Congresses as Topic; D004195:Disease Models, Animal; D004330:Drosophila; D004820:Epidermolysis Bullosa / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy*; D005544:Forecasting; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D038622:Internationality; D051379:Mice; D058990:Molecular Targeted Therapy / Q000379:methods*; D033581:Stem Cell Transplantation / Q000379:methods; D016896:Treatment Outcome; D015027:Zebrafish