Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing

  1. Walker, A.J.
  2. Graham, C.
  3. Greenwood, M.
  4. Woodall, M.
  5. Maeshima, R.
  6. O'Hara-Wright, M.
  7. Sanz, D.J.
  8. Guerrini, I.
  9. Aldossary, A.M.
  10. O'Callaghan, C.
  11. Baines, D.L.
  12. Harrison, P.T.
  13. Hart, S.L.
Revista:
Molecular Therapy - Methods and Clinical Development

ISSN: 2329-0501

Año de publicación: 2023

Volumen: 31

Tipo: Artículo

DOI: 10.1016/J.OMTM.2023.101140 GOOGLE SCHOLAR lock_openAcceso abierto editor