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

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

64 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Messenger RNA therapy for rare genetic metabolic diseases.
Berraondo P, Martini PGV, Avila MA, Fontanellas A. Berraondo P, et al. Among authors: martini pgv. Gut. 2019 Jul;68(7):1323-1330. doi: 10.1136/gutjnl-2019-318269. Epub 2019 Feb 22. Gut. 2019. PMID: 30796097 Review.
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia.
An D, Schneller JL, Frassetto A, Liang S, Zhu X, Park JS, Theisen M, Hong SJ, Zhou J, Rajendran R, Levy B, Howell R, Besin G, Presnyak V, Sabnis S, Murphy-Benenato KE, Kumarasinghe ES, Salerno T, Mihai C, Lukacs CM, Chandler RJ, Guey LT, Venditti CP, Martini PGV. An D, et al. Among authors: martini pgv. Cell Rep. 2017 Dec 19;21(12):3548-3558. doi: 10.1016/j.celrep.2017.11.081. Cell Rep. 2017. PMID: 29262333 Free PMC article.
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. Among authors: martini pgv. Nat Med. 2018 Dec;24(12):1899-1909. doi: 10.1038/s41591-018-0199-z. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297912
mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks.
Jericó D, Córdoba KM, Jiang L, Schmitt C, Morán M, Sampedro A, Alegre M, Collantes M, Santamaría E, Alegre E, Culerier C, de Mendoza AE, Oyarzabal J, Martín MA, Peñuelas I, Ávila MA, Gouya L, Martini PGV, Fontanellas A. Jericó D, et al. Among authors: martini pgv. Mol Ther Nucleic Acids. 2021 May 19;25:207-219. doi: 10.1016/j.omtn.2021.05.010. eCollection 2021 Sep 3. Mol Ther Nucleic Acids. 2021. PMID: 34458006 Free PMC article.
Recombinant porphobilinogen deaminase targeted to the liver corrects enzymopenia in a mouse model of acute intermittent porphyria.
Córdoba KM, Serrano-Mendioroz I, Jericó D, Merino M, Jiang L, Sampedro A, Alegre M, Corrales F, Garrido MJ, Martini PGV, Lanciego JL, Prieto J, Berraondo P, Fontanellas A. Córdoba KM, et al. Among authors: martini pgv. Sci Transl Med. 2022 Jan 12;14(627):eabc0700. doi: 10.1126/scitranslmed.abc0700. Epub 2022 Jan 12. Sci Transl Med. 2022. PMID: 35020410
mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency.
Cao J, An D, Galduroz M, Zhuo J, Liang S, Eybye M, Frassetto A, Kuroda E, Funahashi A, Santana J, Mihai C, Benenato KE, Kumarasinghe ES, Sabnis S, Salerno T, Coughlan K, Miracco EJ, Levy B, Besin G, Schultz J, Lukacs C, Guey L, Finn P, Furukawa T, Giangrande PH, Saheki T, Martini PGV. Cao J, et al. Among authors: martini pgv. Mol Ther. 2019 Jul 3;27(7):1242-1251. doi: 10.1016/j.ymthe.2019.04.017. Epub 2019 Apr 23. Mol Ther. 2019. PMID: 31056400 Free PMC article.
64 results