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 Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

175 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
A framework for individualized splice-switching oligonucleotide therapy.
Kim J, Woo S, de Gusmao CM, Zhao B, Chin DH, DiDonato RL, Nguyen MA, Nakayama T, Hu CA, Soucy A, Kuniholm A, Thornton JK, Riccardi O, Friedman DA, El Achkar CM, Dash Z, Cornelissen L, Donado C, Faour KNW, Bush LW, Suslovitch V, Lentucci C, Park PJ, Lee EA, Patterson A, Philippakis AA, Margus B, Berde CB, Yu TW. Kim J, et al. Among authors: nguyen ma. Nature. 2023 Jul;619(7971):828-836. doi: 10.1038/s41586-023-06277-0. Epub 2023 Jul 12. Nature. 2023. PMID: 37438524 Free PMC article.
Whole Exome Sequencing Is the Preferred Strategy to Identify the Genetic Defect in Patients With a Probable or Possible Mitochondrial Cause.
Theunissen TEJ, Nguyen M, Kamps R, Hendrickx AT, Sallevelt SCEH, Gottschalk RWH, Calis CM, Stassen APM, de Koning B, Mulder-Den Hartog ENM, Schoonderwoerd K, Fuchs SA, Hilhorst-Hofstee Y, de Visser M, Vanoevelen J, Szklarczyk R, Gerards M, de Coo IFM, Hellebrekers DMEI, Smeets HJM. Theunissen TEJ, et al. Front Genet. 2018 Oct 12;9:400. doi: 10.3389/fgene.2018.00400. eCollection 2018. Front Genet. 2018. PMID: 30369941 Free PMC article.
Gene editing without ex vivo culture evades genotoxicity in human hematopoietic stem cells.
Zeng J, Nguyen MA, Liu P, da Silva LF, Levesque S, Lin LY, Justus DG, Petri K, Clement K, Porter SN, Verma A, Neri NR, Rosanwo T, Ciuculescu MF, Abriss D, Mintzer E, Maitland SA, Demirci S, Cha HJ, Orkin SH, Tisdale JF, Williams DA, Zhu LJ, Pruett-Miller SM, Pinello L, Joung JK, Pattanayak V, Manis JP, Armant M, Pellin D, Brendel C, Wolfe SA, Bauer DE. Zeng J, et al. Among authors: nguyen ma. Cell Stem Cell. 2024 Dec 11:S1934-5909(24)00400-4. doi: 10.1016/j.stem.2024.11.001. Online ahead of print. Cell Stem Cell. 2024. PMID: 39672163
BCL11A +58/+55 enhancer-editing facilitates HSPC engraftment and HbF induction in rhesus macaques conditioned with a CD45 antibody-drug conjugate.
Demirci S, Zeng J, Palchaudhuri R, Wu C, Abraham DM, Hayal TB, Essawi K, Nguyen MA, Stasula U, Chu R, Leonard A, Porter SN, Khan MBN, Hinojosa G, Uchida N, Hong S, Lazzarotto CR, Neri NR, da Silva LF, Pellin D, Verma A, Lanieri L, Bhat A, Hammond K, Tate T, Maitland SA, Sheikhsaran F, Bonifacino AC, Krouse AE, Linde NS, Engels T, Golomb J, Tsai SQ, Pruett-Miller SM, Scadden DT, Dunbar CE, Wolfe SA, Donahue RE, Olson LM, Bauer DE, Tisdale JF. Demirci S, et al. Among authors: nguyen ma. Cell Stem Cell. 2024 Dec 5:S1934-5909(24)00376-X. doi: 10.1016/j.stem.2024.10.014. Online ahead of print. Cell Stem Cell. 2024. PMID: 39642886
Corrigendum: An orally available small molecule that targets soluble TNF to deliver anti-TNF biologic-like efficacy in rheumatoid arthritis.
Vugler A, O'connell J, Nguyen MA, Weitz D, Leeuw T, Hickford E, Verbitsky A, Ying X, Rehberg M, Carrington B, Merriman M, Moss A, Nicolas JM, Stanley P, Wright S, Bourne T, Foricher Y, Zhu Z, Brookings D, Horsley H, Heer J, Schio L, Herrmann M, Rao S, Kohlmann M, Florian P. Vugler A, et al. Among authors: nguyen ma. Front Pharmacol. 2024 Oct 16;15:1479557. doi: 10.3389/fphar.2024.1479557. eCollection 2024. Front Pharmacol. 2024. PMID: 39478966 Free PMC article.
Scalable assessment of genome editing off-targets associated with genetic variants.
Lin J, Nguyen MA, Lin LY, Zeng J, Verma A, Neri NR, da Silva LF, Mucci A, Wolfe S, Shaw KL, Clement K, Brendel C, Pinello L, Pellin D, Bauer DE. Lin J, et al. Among authors: nguyen ma. bioRxiv [Preprint]. 2024 Jul 25:2024.07.24.605019. doi: 10.1101/2024.07.24.605019. bioRxiv. 2024. PMID: 39211178 Free PMC article. Preprint.
175 results