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 for this work

Vrije Universiteit Amsterdam
University of Amsterdam
Center for Applied Genomics
Leipzig University
University of Hamburg
Memorial Sloan-Kettering Cancer Center
San Raffaele Telethon Institute for Gene Therapy
SJD Barcelona Children's Hospital
Lund University
University of Copenhagen
University of Tübingen
Utrecht University
Royal Manchester Children's Hospital
Manchester University NHS Foundation Trust
Sorbonne Université
Assistance publique – Hôpitaux de Paris
University of Oslo
German Center for Neurodegenerative Diseases
Tel Aviv University

Research output: Contribution to journal › Article › Academic › peer-review

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Original language	English
Pages (from-to)	12-15
Number of pages	4
Journal	Protein and Cell
Volume	16
Issue number	1
DOIs	https://doi.org/10.1093/procel/pwae065
Publication status	Published - 1 Jan 2025

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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

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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 Jaap-Jan Boelens 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",

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doi = "10.1093/procel/pwae065",

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Schoenmakers, DH , Beerepoot, S, Adang, LA, Asbreuk, MABC, Bergner, CG, Bley, AE, Boelens, J-J, 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, vol. 16, no. 1, pp. 12-15. https://doi.org/10.1093/procel/pwae065

TY - JOUR

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 - Article

SN - 1674-800X

VL - 16

SP - 12

EP - 15

JO - Protein and Cell

JF - Protein and Cell

IS - 1

ER -

Gene therapy in advanced metachromatic leukodystrophy: tempering expectations

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