Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. (Q30541387)

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English	Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.	scientific article

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14 February 2020

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model (English)

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14 February 2020

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mucopolysaccharidosis I

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Letterio S. Politi

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Alessandro Rubinacci

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Riccardo Brambilla

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Angelo Quattrini

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Ilaria Visigalli

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14 February 2020

Stefania Delai

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14 February 2020

Carmela Di Domenico

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14 February 2020

Federica Cerri

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14 February 2020

Emanuela Mrak

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14 February 2020

Daniela Ungaro

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14 February 2020

Merel Stok

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14 February 2020

Francesca Sanvito

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Elisabetta Mariani

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14 February 2020

Lidia Staszewsky

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14 February 2020

Francesca Cecere

15

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14 February 2020

Ubaldo Del Carro

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Paola Di Natale

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Katherine Ponder

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14 February 2020

Alessandra Biffi

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16 September 2010

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14 February 2020

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5130-5139

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14 February 2020

Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

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A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice

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Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

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Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice

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10.1182/BLOOD-2010-04-278234

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14 February 2020

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14 February 2020

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14 February 2020

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