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2018
Mark Fetal gene therapy for neurodegenerative disease of infants
Massaro, Giulia ; Mattar, Citra N.Z. ; Wong, Andrew M.S. ; Sirka, Ernestas ; Buckley, Suzanne M.K. ; Herbert, Bronwen R. ; Karlsson, Stefan ^LU ; Perocheau, Dany P. ; Burke, Derek and Heales, Simon , et al. (2018) In Nature Medicine 24(9). p.1317-1323
- Contribution to journal › Letter
2015
Mark Proteomic Discovery and Development of a Multiplexed Targeted MRM-LC-MS/MS Assay for Urine Biomarkers of Extracellular Matrix Disruption in Mucopolysaccharidoses I, II, and VI
Heywood, Wendy E. ; Camuzeaux, Stephane ; Doykov, Ivan ; Patel, Nina ; Preece, Rhian Lauren ; Footitt, Emma ; Cleary, Maureen ; Clayton, Peter ; Grunewald, Stephanie and Abulhoul, Lara , et al. (2015) In Analytical Chemistry 87(24). p.12238-12244
- Contribution to journal › Article
2013
Mark Increased glucocerebrosidase (GBA) 2 activity in GBA1 deficient mice brains and in Gaucher leucocytes
Burke, Derek G. ; Rahim, Ahad A. ; Waddington, Simon N. ; Karlsson, Stefan ^LU ; Berglin-Enquist, Ida ^LU ; Bhatia, Kailash ; Mehta, Atul ; Vellodi, Ashok and Heales, Simon (2013) In Journal of Inherited Metabolic Disease 36(5). p.869-872
- Contribution to journal › Article