Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome | Zendy

Bernhard Gentner | Zendy; Francesca Tucci | Zendy; Stefania Galimberti | Zendy; Francesca Fumagalli | Zendy; Maurizio De Pellegrin | Zendy; Paolo Silvani | Zendy; Chiara Camesasca | Zendy; Silvia Pontesilli | Zendy; Silvia Darin | Zendy; Francesca Ciotti | Zendy; Marina Sarzana | Zendy; Giulia Consiglieri | Zendy; Chiara Filisetti | Zendy; G Forni | Zendy; Laura Passerini | Zendy; Daniela Tomasoni | Zendy; Daniela Cesana | Zendy; Andrea Calabria | Zendy; Giulio Spinozzi | Zendy; Maria-Pia Cicalese | Zendy; Valeria Calbi | Zendy; Maddalena Migliavacca | Zendy; Federica Barzaghi | Zendy; Francesca Ferrua | Zendy; Vera Gallo | Zendy; Simona Miglietta | Zendy; Erika Zonari | Zendy; Patali S. Cheruku | Zendy; Cristiana Forni | Zendy; Marcella Facchini | Zendy; Ambra Corti | Zendy; Michela Gabaldo | Zendy; Stefano Zancan | Zendy; Serena Gasperini | Zendy; Attilio Rovelli | Zendy; Jaap Jan Boelens | Zendy; Simon Jones | Zendy; Robert Wynn | Zendy; Cristina Baldoli | Zendy; Eugenio Montini | Zendy; Silvia Gregori | Zendy; Fabio Ciceri | Zendy; Maria Grazia Valsecchi | Zendy; Giancarlo la Marca | Zendy; Rossella Parini | Zendy; Luigi Naldini | Zendy; Alessandro Aiuti | Zendy; Maria-Ester Bernardo | Zendy

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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

Author(s) -

Bernhard Gentner,

Francesca Tucci,

Stefania Galimberti,

Francesca Fumagalli,

Maurizio De Pellegrin,

Paolo Silvani,

Chiara Camesasca,

Silvia Pontesilli,

Silvia Darin,

Francesca Ciotti,

Marina Sarzana,

Giulia Consiglieri,

Chiara Filisetti,

G Forni,

Laura Passerini,

Daniela Tomasoni,

Daniela Cesana,

Andrea Calabria,

Giulio Spinozzi,

Maria-Pia Cicalese,

Valeria Calbi,

Maddalena Migliavacca,

Federica Barzaghi,

Francesca Ferrua,

Vera Gallo,

Simona Miglietta,

Erika Zonari,

Patali S. Cheruku,

Cristiana Forni,

Marcella Facchini,

Ambra Corti,

Michela Gabaldo,

Stefano Zancan,

Serena Gasperini,

Attilio Rovelli,

Jaap Jan Boelens,

Simon Jones,

Robert Wynn,

Cristina Baldoli,

Eugenio Montini,

Silvia Gregori,

Fabio Ciceri,

Maria Grazia Valsecchi,

Giancarlo la Marca,

Rossella Parini,

Luigi Naldini,

Alessandro Aiuti,

Maria-Ester Bernardo

Publication year - 2021

Publication title -

new england journal of medicine

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 19.889

H-Index - 1030

eISSN - 1533-4406

pISSN - 0028-4793

DOI - 10.1056/nejmoa2106596

Subject(s) - hurler syndrome , mucopolysaccharidosis type i , hematopoietic stem cell transplantation , progenitor cell , mucopolysaccharidosis i , mucopolysaccharidosis , genetic enhancement , hematopoietic stem cell , stem cell , haematopoiesis , medicine , transplantation , immunology , gene , enzyme replacement therapy , biology , disease , genetics

Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.

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