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Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development
by
Massaro, Giulia
,
Geard, Amy F
,
Liu, Wenfei
,
Coombe-Tennant, Oliver
,
Waddington, Simon N
,
Baruteau, Julien
,
Gissen, Paul
,
Rahim, Ahad A
Published in
Biomolecules (Basel, Switzerland)
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Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease
by
Massaro, Giulia
,
Geard, Amy F
,
Nelvagal, Hemanth R
,
Gore, Katrina
,
Clemo, Nadine K
,
Waddington, Simon N
,
Rahim, Ahad A
Published in
Human molecular genetics
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