Gene therapy with AR isoform 2 rescues spinal and bulbar muscular atrophy phenotype by modulating AR transcriptional activity. uri icon

authors

  • Lim, Wooi F
  • Banerjee, Subhashis
  • Conceição, Mariana
  • Cravo, Lara
  • Biscans, Annabelle
  • Roux, Loïc
  • Pourshafie, Naemeh
  • Grunseich, Christopher
  • Duguez, Stephanie
  • Khvorova, Anastasia
  • Pennuto, Maria
  • Forouhan, Mitra
  • Cortes, Constanza J
  • Al La Spada, MD, PhD
  • Fischbeck, Kenneth H
  • Wood, Matthew J A
  • Rinaldi, Carlo
  • Roberts, Thomas C
  • Dabney, Jesse
  • Ellerington, Ruth
  • Speciale, Alfina A
  • Manzano, Raquel
  • Lieto, Maria
  • Sangha, Gavinda

publication date

  • January 1, 2021