Gene therapy in advanced metachromatic leukodystrophy: tempering expectations

Daphne H. Schoenmakers; Shanice Beerepoot; Laura A. Adang; Marije A.B.C. Asbreuk; Caroline G. Bergner; Annette E. Bley; Jaap Jan Boelens; Valeria Calbi; Alejandra Darling; Erik Eklund; Ángeles García Cazorla; Sabine W. Grønborg; Samuel Groeschel; Peter M. van Hasselt; Carla E.M. Hollak; Claire Horgan; Simon Jones; Tom de Koning; Lucia Laugwitz; Caroline Lindemans; Pascal Martin; Fanny Mochel; Andreas Øberg; Dipak Ram; Caroline Sevin; Ludger Schöls; Ayelet Zerem; Nicole I. Wolf; Francesca Fumagalli

doi:10.1093/procel/pwae065

Gene therapy in advanced metachromatic leukodystrophy: tempering expectations

Daphne H. Schoenmakers^*
, Shanice Beerepoot
, Laura A. Adang
, Marije A.B.C. Asbreuk
, Caroline G. Bergner
, Annette E. Bley
, Jaap Jan Boelens
, Valeria Calbi
, Alejandra Darling
, Erik Eklund
, Ángeles García Cazorla
, Sabine W. Grønborg
, Samuel Groeschel
, Peter M. van Hasselt
, Carla E.M. Hollak
, Claire Horgan
, Simon Jones
, Tom de Koning
, Lucia Laugwitz
, Caroline Lindemans

Pascal Martin, Fanny Mochel, Andreas Øberg, Dipak Ram, Caroline Sevin, Ludger Schöls, Ayelet Zerem, Nicole I. Wolf, Francesca Fumagalli

^*Corresponding author af dette arbejde

4 Citationer (Scopus)

Originalsprog	Engelsk
Tidsskrift	Protein and Cell
Vol/bind	16
Udgave nummer	1
Sider (fra-til)	12-15
Antal sider	4
ISSN	1674-800X
DOI	https://doi.org/10.1093/procel/pwae065
Status	Udgivet - 1 jan. 2025

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10.1093/procel/pwae065Licens: CC BY

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Schoenmakers, D. H., Beerepoot, S., Adang, L. A., Asbreuk, M. A. B. C., Bergner, C. G., Bley, A. E., Boelens, J. J., Calbi, V., Darling, A., Eklund, E., García Cazorla, Á., Grønborg, S. W., Groeschel, S., van Hasselt, P. M., Hollak, C. E. M., Horgan, C., Jones, S., de Koning, T., Laugwitz, L., ... Fumagalli, F. (2025). Gene therapy in advanced metachromatic leukodystrophy: tempering expectations. Protein and Cell, 16(1), 12-15. https://doi.org/10.1093/procel/pwae065

Schoenmakers, DH, Beerepoot, S, Adang, LA, Asbreuk, MABC, Bergner, CG, Bley, AE, Boelens, JJ, Calbi, V, Darling, A, Eklund, E, García Cazorla, Á, Grønborg, SW, Groeschel, S, van Hasselt, PM, Hollak, CEM, Horgan, C, Jones, S, de Koning, T, Laugwitz, L, Lindemans, C, Martin, P, Mochel, F, Øberg, A, Ram, D, Sevin, C, Schöls, L, Zerem, A, Wolf, NI & Fumagalli, F 2025, 'Gene therapy in advanced metachromatic leukodystrophy: tempering expectations', Protein and Cell, bind 16, nr. 1, s. 12-15. https://doi.org/10.1093/procel/pwae065

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title = "Gene therapy in advanced metachromatic leukodystrophy: tempering expectations",

author = "Schoenmakers, \{Daphne H.\} and Shanice Beerepoot and Adang, \{Laura A.\} and Asbreuk, \{Marije A.B.C.\} and Bergner, \{Caroline G.\} and Bley, \{Annette E.\} and Boelens, \{Jaap Jan\} and Valeria Calbi and Alejandra Darling and Erik Eklund and \{Garc{\'i}a Cazorla\}, {\'A}ngeles and Gr{\o}nborg, \{Sabine W.\} and Samuel Groeschel and \{van Hasselt\}, \{Peter M.\} and Hollak, \{Carla E.M.\} and Claire Horgan and Simon Jones and \{de Koning\}, Tom and Lucia Laugwitz and Caroline Lindemans and Pascal Martin and Fanny Mochel and Andreas {\O}berg and Dipak Ram and Caroline Sevin and Ludger Sch{\"o}ls and Ayelet Zerem and Wolf, \{Nicole I.\} and Francesca Fumagalli",

year = "2025",

month = jan,

day = "1",

doi = "10.1093/procel/pwae065",

language = "English",

volume = "16",

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T1 - Gene therapy in advanced metachromatic leukodystrophy

T2 - tempering expectations

AU - Schoenmakers, Daphne H.

AU - Beerepoot, Shanice

AU - Adang, Laura A.

AU - Asbreuk, Marije A.B.C.

AU - Bergner, Caroline G.

AU - Bley, Annette E.

AU - Boelens, Jaap Jan

AU - Calbi, Valeria

AU - Darling, Alejandra

AU - Eklund, Erik

AU - García Cazorla, Ángeles

AU - Grønborg, Sabine W.

AU - Groeschel, Samuel

AU - van Hasselt, Peter M.

AU - Hollak, Carla E.M.

AU - Horgan, Claire

AU - Jones, Simon

AU - de Koning, Tom

AU - Laugwitz, Lucia

AU - Lindemans, Caroline

AU - Martin, Pascal

AU - Mochel, Fanny

AU - Øberg, Andreas

AU - Ram, Dipak

AU - Sevin, Caroline

AU - Schöls, Ludger

AU - Zerem, Ayelet

AU - Wolf, Nicole I.

AU - Fumagalli, Francesca

PY - 2025/1/1

Y1 - 2025/1/1

UR - https://www.scopus.com/pages/publications/85216890630

U2 - 10.1093/procel/pwae065

DO - 10.1093/procel/pwae065

M3 - Journal article

AN - SCOPUS:85216890630

SN - 1674-800X

VL - 16

SP - 12

EP - 15

JO - Protein and Cell

JF - Protein and Cell

IS - 1

ER -