Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Merel Stok, Helen Boer, MW Huston, EH Jacobs, Onno Roovers, Trui Visser, Holger Jahr, Dirk-jan Duncker, Elza Deel, Arnold Reuser, Niek Til, Gerard Wagemaker

Research output: Contribution to journalArticleAcademicpeer-review

30 Citations (Scopus)
39 Downloads (Pure)
Original languageEnglish
Pages (from-to)1014-1025
Number of pages12
JournalMolecular Therapy-Methods & Clinical Development
Volume17
DOIs
Publication statusPublished - 2020

Research programs

  • EMC MGC-01-12-03
  • EMC OR-01
  • EMC MM-01-54-01

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