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.
Journal:
Molecular Therapy

ISSN: 1525-0024 1525-0016

Year of publication: 2024

Type: Article

DOI: 10.1016/J.YMTHE.2024.03.009 GOOGLE SCHOLAR