Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, Maria Sessa, Cristina Baldoli, Paola Maria V Rancoita, Francesca Ciotti, Marina Sarzana, Maddalena Fraschini, Alberto Andrea Zambon, Serena Acquati, Daniela Redaelli, Vanessa Attanasio, Simona Miglietta, Fabiola De Mattia, Federica Barzaghi, Francesca Ferrua, Maddalena Migliavacca, Francesca Tucci, Vera Gallo, Ubaldo Del Carro, Sabrina Canale, Ivana Spiga, Laura Lorioli, Salvatore Recupero, Elena Sophia Fratini, Francesco Morena, Paolo Silvani, Maria Rosa Calvi, Marcella Facchini, Sara Locatelli, Ambra Corti, Stefano Zancan, Gigliola Antonioli, Giada Farinelli, Michela Gabaldo, Jesus Garcia-Segovia, Laetitia C Schwab, Gerald F Downey, Massimo Filippi, Maria Pia Cicalese, Sabata Martino, Clelia Di Serio, Fabio Ciceri, Maria Ester Bernardo, Luigi Naldini, Alessandra Biffi, Alessandro Aiuti

Open source

DOI: 10.1016/s0140-6736(21)02017-1
Published: 2022-01
Container: The Lancet
Publisher: Elsevier BV
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/s0140-6736-21-02017-1,
  title = {Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access},
  author = {Francesca Fumagalli and Valeria Calbi and Maria Grazia Natali Sora and Maria Sessa and Cristina Baldoli and Paola Maria V Rancoita and Francesca Ciotti and Marina Sarzana and Maddalena Fraschini and Alberto Andrea Zambon and Serena Acquati and Daniela Redaelli and Vanessa Attanasio and Simona Miglietta and Fabiola De Mattia and Federica Barzaghi and Francesca Ferrua and Maddalena Migliavacca and Francesca Tucci and Vera Gallo and Ubaldo Del Carro and Sabrina Canale and Ivana Spiga and Laura Lorioli and Salvatore Recupero and Elena Sophia Fratini and Francesco Morena and Paolo Silvani and Maria Rosa Calvi and Marcella Facchini and Sara Locatelli and Ambra Corti and Stefano Zancan and Gigliola Antonioli and Giada Farinelli and Michela Gabaldo and Jesus Garcia-Segovia and Laetitia C Schwab and Gerald F Downey and Massimo Filippi and Maria Pia Cicalese and Sabata Martino and Clelia Di Serio and Fabio Ciceri and Maria Ester Bernardo and Luigi Naldini and Alessandra Biffi and Alessandro Aiuti},
  year = {2022},
  journal = {The Lancet},
  doi = {10.1016/s0140-6736(21)02017-1},
  url = {https://doi.org/10.1016/s0140-6736(21)02017-1}
}

RIS

TY  - JOUR
TI  - Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
AU  - Francesca Fumagalli
AU  - Valeria Calbi
AU  - Maria Grazia Natali Sora
AU  - Maria Sessa
AU  - Cristina Baldoli
AU  - Paola Maria V Rancoita
AU  - Francesca Ciotti
AU  - Marina Sarzana
AU  - Maddalena Fraschini
AU  - Alberto Andrea Zambon
AU  - Serena Acquati
AU  - Daniela Redaelli
AU  - Vanessa Attanasio
AU  - Simona Miglietta
AU  - Fabiola De Mattia
AU  - Federica Barzaghi
AU  - Francesca Ferrua
AU  - Maddalena Migliavacca
AU  - Francesca Tucci
AU  - Vera Gallo
AU  - Ubaldo Del Carro
AU  - Sabrina Canale
AU  - Ivana Spiga
AU  - Laura Lorioli
AU  - Salvatore Recupero
AU  - Elena Sophia Fratini
AU  - Francesco Morena
AU  - Paolo Silvani
AU  - Maria Rosa Calvi
AU  - Marcella Facchini
AU  - Sara Locatelli
AU  - Ambra Corti
AU  - Stefano Zancan
AU  - Gigliola Antonioli
AU  - Giada Farinelli
AU  - Michela Gabaldo
AU  - Jesus Garcia-Segovia
AU  - Laetitia C Schwab
AU  - Gerald F Downey
AU  - Massimo Filippi
AU  - Maria Pia Cicalese
AU  - Sabata Martino
AU  - Clelia Di Serio
AU  - Fabio Ciceri
AU  - Maria Ester Bernardo
AU  - Luigi Naldini
AU  - Alessandra Biffi
AU  - Alessandro Aiuti
PY  - 2022
JO  - The Lancet
DO  - 10.1016/s0140-6736(21)02017-1
UR  - https://doi.org/10.1016/s0140-6736(21)02017-1
ER  -

APA

Fumagalli, F., Calbi, V., Sora, M. G. N., Sessa, M., Baldoli, C., Rancoita, P. M. V., Ciotti, F., Sarzana, M., Fraschini, M., Zambon, A. A., Acquati, S., Redaelli, D., Attanasio, V., Miglietta, S., Mattia, F. D., Barzaghi, F., Ferrua, F., Migliavacca, M., Tucci, F., Gallo, V., Carro, U. D., Canale, S., Spiga, I., Lorioli, L., Recupero, S., Fratini, E. S., Morena, F., Silvani, P., Calvi, M. R., Facchini, M., Locatelli, S., Corti, A., Zancan, S., Antonioli, G., Farinelli, G., Gabaldo, M., Garcia-Segovia, J., Schwab, L. C., Downey, G. F., Filippi, M., Cicalese, M. P., Martino, S., Serio, C. D., Ciceri, F., Bernardo, M. E., Naldini, L., Biffi, A., & Aiuti, A. (2022). Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access. The Lancet. https://doi.org/10.1016/s0140-6736(21)02017-1

Source records

crossref · retrieved 2026-09-26T23:36:47.197Z