UX701 is an experimental gene therapy for Wilson disease, delivered via adeno-associated virus, that restores a functional version of the ATP7B gene.[1][2][3]

References

  1. ↑ Mazhar, Areej; Piper, Marc S. (October 2023). "Updates on Wilson disease". Clinical Liver Disease. 22 (4): 117. doi:10.1097/CLD.0000000000000079. ISSN 2046-2484. PMC 10615495. PMID 37908869.
  2. ↑ Zheng, Zi-Wei; Dong, Yi; Wu, Zhi-Ying (September 2022). "Latest innovations in the treatment of Wilson's disease". ILIVER. 1 (3): 181–186. doi:10.1016/j.iliver.2022.09.002.
  3. ↑ Cataldo, Jason; Allen, Jack; Sankoh, Serap; Weiss, Karl; Askari, Frederick (March 2022). "eP140: A novel, double-blind placebo-controlled seamless phase 1/2/3 AAV9 gene therapy study for Wilson disease". Genetics in Medicine. 24 (3): S86. doi:10.1016/j.gim.2022.01.176.
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