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

131 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.
Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ. Métais JY, et al. Among authors: ryu by. Blood Adv. 2019 Nov 12;3(21):3379-3392. doi: 10.1182/bloodadvances.2019000820. Blood Adv. 2019. PMID: 31698466 Free PMC article.
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Mamcarz E, et al. Among authors: ryu by. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. N Engl J Med. 2019. PMID: 30995372 Free PMC article. Clinical Trial.
Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes.
Ozog S, Timberlake ND, Hermann K, Garijo O, Haworth KG, Shi G, Glinkerman CM, Schefter LE, D'Souza S, Simpson E, Sghia-Hughes G, Carillo RR, Boger DL, Kiem HP, Slukvin I, Ryu BY, Sorrentino BP, Adair JE, Snyder SA, Compton AA, Torbett BE. Ozog S, et al. Among authors: ryu by. Blood. 2019 Oct 17;134(16):1298-1311. doi: 10.1182/blood.2019000040. Blood. 2019. PMID: 31416800 Free PMC article.
Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor.
Uchida N, Ferrara F, Drysdale CM, Yapundich M, Gamer J, Nassehi T, DiNicola J, Shibata Y, Wielgosz M, Kim YS, Bauler M, Throm RE, Haro-Mora JJ, Demirci S, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ryu B, Tisdale JF. Uchida N, et al. Sci Transl Med. 2021 Apr 28;13(591):eabb0411. doi: 10.1126/scitranslmed.abb0411. Sci Transl Med. 2021. PMID: 33910976
Forced enhancer-promoter rewiring to alter gene expression in animal models.
Peslak SA, Demirci S, Chandra V, Ryu B, Bhardwaj SK, Jiang J, Rupon JW, Throm RE, Uchida N, Leonard A, Essawi K, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ferrara F, Wielgosz M, Abdulmalik O, Hamagami N, Germino-Watnick P, Le A, Chu R, Hinds M, Weiss MJ, Tong W, Tisdale JF, Blobel GA. Peslak SA, et al. Mol Ther Nucleic Acids. 2023 Jan 31;31:452-465. doi: 10.1016/j.omtn.2023.01.016. eCollection 2023 Mar 14. Mol Ther Nucleic Acids. 2023. PMID: 36852088 Free PMC article.
131 results