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Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
Everette KA, Newby GA, Levine RM, Mayberry K, Jang Y, Mayuranathan T, Nimmagadda N, Dempsey E, Li Y, Bhoopalan SV, Liu X, Davis JR, Nelson AT, Chen PJ, Sousa AA, Cheng Y, Tisdale JF, Weiss MJ, Yen JS, Liu DR. Everette KA, et al. Among authors: levine rm. Nat Biomed Eng. 2023 May;7(5):616-628. doi: 10.1038/s41551-023-01026-0. Epub 2023 Apr 17. Nat Biomed Eng. 2023. PMID: 37069266 Free PMC article.
Potent and uniform fetal hemoglobin induction via base editing.
Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS. Mayuranathan T, et al. Among authors: levine rm. Nat Genet. 2023 Jul;55(7):1210-1220. doi: 10.1038/s41588-023-01434-7. Epub 2023 Jul 3. Nat Genet. 2023. PMID: 37400614 Free PMC article.
Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.
Lian X, Chatterjee S, Sun Y, Dilliard SA, Moore S, Xiao Y, Bian X, Yamada K, Sung YC, Levine RM, Mayberry K, John S, Liu X, Smith C, Johnson LT, Wang X, Zhang CC, Liu DR, Newby GA, Weiss MJ, Yen JS, Siegwart DJ. Lian X, et al. Among authors: levine rm. Nat Nanotechnol. 2024 Sep;19(9):1409-1417. doi: 10.1038/s41565-024-01680-8. Epub 2024 May 23. Nat Nanotechnol. 2024. PMID: 38783058
Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34+ cell therapy to induce fetal hemoglobin for sickle cell disease.
Katta V, O'Keefe K, Li Y, Mayuranathan T, Lazzarotto CR, Wood RK, Levine RM, Powers A, Mayberry K, Manquen G, Yao Y, Zhang J, Jang Y, Nimmagadda N, Dempsey EA, Lee G, Uchida N, Cheng Y, Fazio F, Lockey T, Meagher M, Sharma A, Tisdale JF, Zhou S, Yen JS, Weiss MJ, Tsai SQ. Katta V, et al. Among authors: levine rm. Mol Ther. 2024 Oct 2;32(10):3433-3452. doi: 10.1016/j.ymthe.2024.07.022. Epub 2024 Jul 31. Mol Ther. 2024. PMID: 39086133 Free article.
Automated real-world data integration improves cancer outcome prediction.
Jee J, Fong C, Pichotta K, Tran TN, Luthra A, Waters M, Fu C, Altoe M, Liu SY, Maron SB, Ahmed M, Kim S, Pirun M, Chatila WK, de Bruijn I, Pasha A, Kundra R, Gross B, Mastrogiacomo B, Aprati TJ, Liu D, Gao J, Capelletti M, Pekala K, Loudon L, Perry M, Bandlamudi C, Donoghue M, Satravada BA, Martin A, Shen R, Chen Y, Brannon AR, Chang J, Braunstein L, Li A, Safonov A, Stonestrom A, Sanchez-Vela P, Wilhelm C, Robson M, Scher H, Ladanyi M, Reis-Filho JS, Solit DB, Jones DR, Gomez D, Yu H, Chakravarty D, Yaeger R, Abida W, Park W, O'Reilly EM, Garcia-Aguilar J, Socci N, Sanchez-Vega F, Carrot-Zhang J, Stetson PD, Levine R, Rudin CM, Berger MF, Shah SP, Schrag D, Razavi P, Kehl KL, Li BT, Riely GJ, Schultz N; MSK Cancer Data Science Initiative Group. Jee J, et al. Among authors: levine r. Nature. 2024 Nov 6. doi: 10.1038/s41586-024-08167-5. Online ahead of print. Nature. 2024. PMID: 39506116
Isolated prolapse of the posterior mitral valve leaflet: phenotypic refinement, heritability and genetic etiology.
Rimbert A, Duval D, Trujillano D, Kyndt F, Jobbe-Duval A, Lindenbaum P, Tucker N, Lecointe S, Labbé P, Toquet C, Karakachoff M, Roussel JC, Baufreton C, Bruneval P, Cueff C, Donal E, Redon R, Olaso R, Boland A, Deleuze JF; FranceGenRef Consortium; Estivill X, Slaugenhaupt S, Markwald RR, Norris RA, Verhoye JP, Probst V, Hagège A, Levine R, Jeunemaitre X, Marec HL, Capoulade R, Bouatia-Naji N, Dina C, Milan D, Ossowski S, Schott JJ, Mérot J, Scouarnec SL, Tourneau TL. Rimbert A, et al. Among authors: levine r. medRxiv [Preprint]. 2024 Oct 17:2024.10.16.24315096. doi: 10.1101/2024.10.16.24315096. medRxiv. 2024. PMID: 39484266 Free PMC article. Preprint.
3,595 results