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2,221 articles

Articles

Original Research

Published on 21 Jul 2023

Expanding the phenotype of THRB: a range of macular dystrophies as the major clinical manifestations in patients with a dominant splicing variant

in Molecular and Cellular Pathology

  • Elena Fernández-Suárez
  • María González-del Pozo
  • Alejandro García-Núñez
  • Cristina Méndez-Vidal
  • Marta Martín-Sánchez
  • José Manuel Mejías-Carrasco
  • Manuel Ramos-Jiménez
  • María José Morillo-Sánchez
  • Enrique Rodríguez-de la Rúa
  • Salud Borrego
Expanding the phenotype of THRB: a range of macular dystrophies as the major clinical manifestations in patients with a dominant splicing variant
Frontiers in Cell and Developmental Biology
doi 10.3389/fcell.2023.1197744
  • 2,069 views
  • 4 citations

Original Research

Published on 11 Jul 2023

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

in Molecular and Cellular Pathology

  • Jerry R. Mendell
  • Perry B. Shieh
  • Craig M. McDonald
  • Zarife Sahenk
  • Kelly J. Lehman
  • Linda P. Lowes
  • Natalie F. Reash
  • Megan A. Iammarino
  • Lindsay N. Alfano
  • Brenna Sabo
Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy
Frontiers in Cell and Developmental Biology
doi 10.3389/fcell.2023.1167762
  • 15,962 views
  • 27 citations