In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice

  1. Böck, D.
  2. Revers, I.M.
  3. Bomhof, A.S.J.
  4. Hillen, A.E.J.
  5. Boeijink, C.
  6. Kissling, L.
  7. Egli, S.
  8. Moreno-Mateos, M.A.
  9. van der Knaap, M.S.
  10. van Til, N.P.
  11. Schwank, G.
Zeitschrift:
Molecular Therapy

ISSN: 1525-0024 1525-0016

Datum der Publikation: 2024

Art: Artikel

DOI: 10.1016/J.YMTHE.2024.03.009 GOOGLE SCHOLAR