Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Comment
. 2019 Sep;70(3):1061-1063.
doi: 10.1002/hep.30678. Epub 2019 May 23.

Acute Intermittent Porphyria: Novel Etiologic and Pathogenic Therapies Based on RNA Transfer to the Liver

Jesus Prieto  1 , Gloria Gonzalez-Aseguinolaza  1
Affiliations
Comment

Acute Intermittent Porphyria: Novel Etiologic and Pathogenic Therapies Based on RNA Transfer to the Liver

Jesus Prieto et al. Hepatology. 2019 Sep.
No abstract available

PubMed Disclaimer

Comment on

  • Systemic messenger RNA as an etiological treatment for acute intermittent porphyria.
    Jiang L, Berraondo P, Jericó D, Guey LT, Sampedro A, Frassetto A, Benenato KE, Burke K, Santamaría E, Alegre M, Pejenaute Á, Kalariya M, Butcher W, Park JS, Zhu X, Sabnis S, Kumarasinghe ES, Salerno T, Kenney M, Lukacs CM, Ávila MA, Martini PGV, Fontanellas A. Jiang L, et al. Nat Med. 2018 Dec;24(12):1899-1909. doi: 10.1038/s41591-018-0199-z. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297912
  • Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
    Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. Sardh E, et al. N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838. N Engl J Med. 2019. PMID: 30726693 Clinical Trial.

References

    1. Bissell DM, Anderson KE, Bonkovsky HL. Porphyria. N Engl J Med 2017;377:862-872.
    1. D'Avola D, López-Franco E, Sangro B, Pañeda A, Grossios N, Gil-Farina I, et al. Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria. J Hepatol 2016;65:776-778.
    1. Jiang L, Berraondo P, Jericó D, Guey LT, Sampedro A, Frassetto A, et al. Systemic messenger RNA as an etiological treatment for acute intermittent porphyria. Nat Med 2018;24:1899-1909.
    1. Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, et al. Phase 1 trial of an RNA interference therapy for acute intermittent porphyria. N Engl J Med 2019;380:549-558.
    1. Ahmed A, Rahman M, Zhang X. Induction of placental heme oxygenase-1 is protective against TNFalpha induced cytotoxicity and promotes vessel relaxation. Mol Med 2000;6:391-409.

Publication types

LinkOut - more resources