Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.

Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell'Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, Brunetti-Pierri R, Simonelli F, Nigro V, Vargas M, Servillo G, Borgia F, Soscia E, Gargaro M, Funghini S, Tedesco N, Le Brun PR, Rupar CA, Prasad C, O'Callaghan M, Mitchell JJ, Danos O, Marteau JB, Galimberti S, Valsecchi MG, Veron P, Mingozzi F, Fallarino F, la Marca G, Sivri HS, Auricchio A

Open source

DOI: 10.1056/evidoa2200052
Published: 2022 Jul
Container: NEJM evidence
Publisher: Not recorded
Open access: no

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Cite this work

BibTeX

@article{allodium:10.1056/evidoa2200052,
  title = {Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.},
  author = {Brunetti-Pierri N and Ferla R and Ginocchio VM and Rossi A and Fecarotta S and Romano R and Parenti G and Yildiz Y and Zancan S and Pecorella V and Dell'Anno M and Graziano M and Alliegro M and Andria G and Santamaria F and Brunetti-Pierri R and Simonelli F and Nigro V and Vargas M and Servillo G and Borgia F and Soscia E and Gargaro M and Funghini S and Tedesco N and Le Brun PR and Rupar CA and Prasad C and O'Callaghan M and Mitchell JJ and Danos O and Marteau JB and Galimberti S and Valsecchi MG and Veron P and Mingozzi F and Fallarino F and la Marca G and Sivri HS and Auricchio A},
  year = {2022},
  journal = {NEJM evidence},
  doi = {10.1056/evidoa2200052},
  url = {https://doi.org/10.1056/evidoa2200052}
}

RIS

TY  - JOUR
TI  - Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.
AU  - Brunetti-Pierri N
AU  - Ferla R
AU  - Ginocchio VM
AU  - Rossi A
AU  - Fecarotta S
AU  - Romano R
AU  - Parenti G
AU  - Yildiz Y
AU  - Zancan S
AU  - Pecorella V
AU  - Dell'Anno M
AU  - Graziano M
AU  - Alliegro M
AU  - Andria G
AU  - Santamaria F
AU  - Brunetti-Pierri R
AU  - Simonelli F
AU  - Nigro V
AU  - Vargas M
AU  - Servillo G
AU  - Borgia F
AU  - Soscia E
AU  - Gargaro M
AU  - Funghini S
AU  - Tedesco N
AU  - Le Brun PR
AU  - Rupar CA
AU  - Prasad C
AU  - O'Callaghan M
AU  - Mitchell JJ
AU  - Danos O
AU  - Marteau JB
AU  - Galimberti S
AU  - Valsecchi MG
AU  - Veron P
AU  - Mingozzi F
AU  - Fallarino F
AU  - la Marca G
AU  - Sivri HS
AU  - Auricchio A
PY  - 2022
JO  - NEJM evidence
DO  - 10.1056/evidoa2200052
UR  - https://doi.org/10.1056/evidoa2200052
ER  -

APA

N, B., R, F., VM, G., A, R., S, F., R, R., G, P., Y, Y., S, Z., V, P., M, D., M, G., M, A., G, A., F, S., R, B., F, S., V, N., M, V., G, S., F, B., E, S., M, G., S, F., N, T., PR, L. B., CA, R., C, P., M, O., JJ, M., O, D., JB, M., S, G., MG, V., P, V., F, M., F, F., G, L. M., HS, S., & A, A. (2022). Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.. NEJM evidence. https://doi.org/10.1056/evidoa2200052

Source records

pubmed · retrieved 2026-09-25T08:28:46.880Z
europe-pmc · retrieved 2026-09-25T08:28:47.339Z