34504088

Summary
Title
Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy.
Authors
  • Bertolin J
  • Sánchez V
  • Ribera A
  • Jaén ML
  • Garcia M
  • Pujol A
  • Sánchez X
  • Muñoz S
  • Marcó S
  • Pérez J
  • Elias G
  • León X
  • Roca C
  • Jimenez V
  • Otaegui P
  • Mulero F
  • Navarro M
  • Ruberte J
  • Bosch F
Source
Nat Commun
Publication Date
2021 Sep 9
PubMed ID
34504088

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Acknowledgements

Supported by JST NBDC Grant Number JPMJND2204

Partly supported by NIH Common Fund Grant #1U01GM125267-01


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