Interim non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy for Mucopolysaccharidosis type I Hurler

被引：0

作者：

Tucci, F. ^{[1
]}

Filisetti, C. ^{[1
]}

Cossutta, M. ^{[1
]}

Consiglieri, G. ^{[1
]}

Galimberti, L. ^{[1
]}

Catalli, B. ^{[1
]}

Fumagalli, F. ^{[1
]}

Butera, C. ^{[1
]}

Santangelo, R. ^{[1
]}

Colombo, M. ^{[1
]}

Camesasca, C. ^{[1
]}

Darin, S. ^{[1
]}

Facchini, M. ^{[1
]}

Albertazzi, E. ^{[1
]}

Ciceri, F. ^{[1
]}

Naldini, L. ^{[1
]}

Parini, R. ^{[1
]}

Gentner, B. ^{[1
]}

Aiuti, A. ^{[1
]}

Bernardo, M. E. ^{[1
]}

机构：

[1] San Raffaele Telethon Inst Gene Therapy, HSR TIGET, Milan, Italy

来源：

HUMAN GENE THERAPY | 2024年 / 35卷 / 3-4期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

P560

引用

页码：A240 / A240

页数：1

共 34 条

[1] NON-NEUROLOGICAL, NON-SKELETAL OUTCOMES AFTER HAEMATOPOIETIC STEM AND PROGENITOR CELL-GENE THERAPY IN HURLER PATIENTS: RETROSPECTIVE COMPARISON WITH ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANTATION
Tucci, Francesca
Uria Oficialdegui, Maria Luz
Consiglieri, Giulia
Cossutta, Matilde
Filisetti, Chiara
Galimberti, Luca
Spiridon, Mariana Priviero
Manitto, Maria Pia
Butera, Carla
Santangelo, Roberto
Dane, Giulia
Camesasca, Chiara
Darin, Silvia
Del Toro, Mireia
de Hereida Rubio, Cristina Diaz
Parini, Rossella
Gentner, Bernhard
Aiuti, Alessandro
Bernardo, Maria Ester
BONE MARROW TRANSPLANTATION, 2024, 59 : 66 - 67
[2] Non-Neurological, Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene Therapy in Hurler Patients: Retrospective Comparison with Allogeneic Hematopoietic Stem Cell Transplantation
Tucci, Francesca
Uria Oficialdegui, Maria Luz
Consiglieri, Giulia
Cossutta, Matilde
Filisetti, Chiara
Galimberti, Luca
Spiridon, Mariana Priviero
Manitto, Maria Pia
Butera, Carla
Santangelo, Roberto
Dane, Giulia
Camesasca, Chiara
Darin, Silvia
Del Toro, Mireia
Diaz de Hereida, Cristina
Parini, Rossella
Aiuti, Alessandro
Bernardo, Maria Ester
MOLECULAR THERAPY, 2024, 32 (04) : 886 - 886
[3] Early Skeletal Outcome after Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type I Hurler
De Pellegrin, Maurizio
Consiglieri, Giulia
Tucci, Francesca
Guerrini, Barbara
Pontesilli, Silvia
Filisetti, Chiara
Scarparo, Stefano
Sarzana, Marina
Silvani, Paolo
Forni, Giulia
Miglietta, Simona
Gasperini, Serena
Cattoni, Alessandro
Baldoli, Cristina
Ciceri, Fabio
La Marca, Giancarlo
Parini, Rossella
Naldini, Luigi M.
Aiuti, Alessandro
Gentner, Bernhard Rudolf
Bernardo, Maria Ester
MOLECULAR THERAPY, 2023, 31 (04) : 3 - 3
[4] Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome
Consiglieri, Giulia
Tucci, Francesca
De Pellegrin, Maurizio
Guerrini, Barbara
Cattoni, Alessandro
Risca, Giulia
Scarparo, Stefano
Sarzana, Marina
Pontesilli, Silvia
Mellone, Renata
Gasperini, Serena
Galimberti, Stefania
Silvani, Paolo
Filisetti, Chiara
Darin, Silvia
Forni, Giulia
Miglietta, Simona
Santi, Ludovica
Facchini, Marcella
Corti, Ambra
Fumagalli, Francesca
Cicalese, Maria Pia
Calbi, Valeria
Migliavacca, Maddalena
Barzaghi, Federica
Ferrua, Francesca
Gallo, Vera
Recupero, Salvatore
Canarutto, Daniele
Doglio, Matteo
Tedesco, Lucia
Volpi, Nicola
Rovelli, Attilio
la Marca, Giancarlo
Valsecchi, Maria Grazia
Zancan, Stefano
Ciceri, Fabio
Naldini, Luigi
Baldoli, Cristina
Parini, Rossella
Gentner, Bernhard
Aiuti, Alessandro
Bernardo, Maria Ester
SCIENCE TRANSLATIONAL MEDICINE, 2024, 16 (745)
[5] Hematopoietic stem cell gene therapy for mucopolysaccharidosis type I-Hurler syndrome (OTL-203): Interim skeletal, neurological and systemic outcomes from a phase I/II study
Bernardo, Maria Ester M.
Tucci, Francesca
Consiglieri, Giulia
Oficialdegui, Maria Luz Uria
Fumagalli, Francesca
De Pellegrin, Maurizio
Cossutta, Matilde
Filisetti, Chiara
Manitto, Maria Pia
Butera, Carla
Dane, Giulia
Camesasca, Chiara
Parini, Rossella
Riera, Mireia Del Toro
de Heredia, Cristina Diaz
Aiuti, Alessandro
MOLECULAR GENETICS AND METABOLISM, 2025, 144 (02) : 10 - 11
[6] Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome)
Gentner, Bernhard
Bernardo, Maria Ester
Tucci, Francesca
Fumagalli, Francesca
Pontesilli, Silvia
Silvani, Paolo
Zonari, Erika
Miglietta, Simona
Montini, Eugenio
Ciceri, Fabio
La Marca, Giancarlo
Parini, Rossella
Naldini, Luigi
Aiuti, Alessandro
MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S42 - S43
[7] Therapeutic efficacy of hematopoietic stem cell gene therapy for Hurler Type 1 Mucopolysaccharidosis
Delai, S.
Visigalli, I.
Politi, Ls
Mrak, E.
Di Domenico, C.
Mariani, E.
Stok, M.
Del Carro, U.
Rubinacci, A.
Brambilla, R.
Quattrini, A.
Di Natale, P.
Ponder, K.
Naldini, L.
Biffi, A.
HUMAN GENE THERAPY, 2010, 21 (10) : 1436 - 1437
[8] Therapeutic Efficacy of Hematopoietic Stem Cell Gene Therapy for Hurler Type 1 Mucopolysaccharidosis
Visigalli, Ilaria
Delai, Stefania
Stok, Merel
Cesani, Martina
Plati, Tiziana
Politi, Letterio S.
Di Natale, Paola
Latini, Roberto
Staszewski, Lidia I.
Brambilla, Roberto
Quattrini, Angelo
Mariani, Elisabetta
Cenci, Simone
Mrak, Emanuela
Rubinacci, Alessandro
Sanvito, Francesca
Ponzoni, Maurilio
Naldini, Luigi
Biffi, Alessandra
MOLECULAR THERAPY, 2009, 17 : S340 - S340
[9] Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
Bertolin, Joan
Sanchez, Victor
Ribera, Albert
Jaen, Maria Luisa
Garcia, Miquel
Pujol, Anna
Sanchez, Xavier
Munoz, Sergio
Marco, Sara
Perez, Jennifer
Elias, Gemma
Leon, Xavier
Roca, Carles
Jimenez, Veronica
Otaegui, Pedro
Mulero, Francisca
Navarro, Marc
Ruberte, Jesus
Bosch, Fatima
NATURE COMMUNICATIONS, 2021, 12 (01)
[10] Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
Joan Bertolin
Víctor Sánchez
Albert Ribera
Maria Luisa Jaén
Miquel Garcia
Anna Pujol
Xavier Sánchez
Sergio Muñoz
Sara Marcó
Jennifer Pérez
Gemma Elias
Xavier León
Carles Roca
Veronica Jimenez
Pedro Otaegui
Francisca Mulero
Marc Navarro
Jesús Ruberte
Fatima Bosch
Nature Communications, 12

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