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

8 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency.
Khoja S, Liu XB, Truong B, Nitzahn M, Lambert J, Eliav A, Nasser E, Randolph E, Burke KE, White R, Zhu X, Martini PGV, Nissim I, Cederbaum SD, Lipshutz GS. Khoja S, et al. Among authors: eliav a. Mol Ther Nucleic Acids. 2022 Apr 27;28:859-874. doi: 10.1016/j.omtn.2022.04.012. eCollection 2022 Jun 14. Mol Ther Nucleic Acids. 2022. PMID: 35694211 Free PMC article.
Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities.
Khoja S, Lambert J, Nitzahn M, Eliav A, Zhang Y, Tamboline M, Le CT, Nasser E, Li Y, Patel P, Zhuravka I, Lueptow LM, Tkachyova I, Xu S, Nissim I, Schulze A, Lipshutz GS. Khoja S, et al. Among authors: eliav a. Mol Ther Methods Clin Dev. 2022 Mar 28;25:278-296. doi: 10.1016/j.omtm.2022.03.015. eCollection 2022 Jun 9. Mol Ther Methods Clin Dev. 2022. PMID: 35505663 Free PMC article.