Zobrazeno 1 - 1
of 1
pro vyhledávání: '"MESH: Mucopolysaccharidosis III* / therapy"'
Autor:
Jérôme Ausseil, Béatrice Poirier-Beaudouin, Marie-Lise Gougeon, Béatrice Husson, Dimitrios I. Zafeiriou, Marc Tardieu, Giancarlo Parenti, Kumaran Deiva, Michel Zerah, Stéphanie de Bournonville, Jean-Michel Heard
Publikováno v:
Human Gene Therapy
Human Gene Therapy, 2021, 32 (19-20), pp.1251-1259. ⟨10.1089/hum.2021.135⟩
Human Gene Therapy, 2021, 32 (19-20), pp.1251-1259. ⟨10.1089/hum.2021.135⟩
International audience; We report the safety (primary endpoint) and efficacy (secondary endpoint) of a novel intracerebral gene therapy at 5.5 years of follow-up in children with Sanfilippo B. An uncontrolled, phase 1/2 clinical trial was performed i
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5656e00806ab496dfb15526727215b31
http://hdl.handle.net/11588/870281
http://hdl.handle.net/11588/870281