Original paper

Genetically Modulated Substrate Reduction Therapy for Sanfilippo syndrome – proof of principle

Published: Nov 1, 2017
Abstract
This work was supported by Fundacao Millennium bcp (bcp/LIM/DGH/2014). JIS and MFC are grantees from the FCT (SFRH/BD/124372/2016 and...
Paper Details
Title
Genetically Modulated Substrate Reduction Therapy for Sanfilippo syndrome – proof of principle
Published Date
Nov 1, 2017
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