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2003 2
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64 results

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Page 1
In vivo hematopoietic stem cell modification by mRNA delivery.
Breda L, Papp TE, Triebwasser MP, Yadegari A, Fedorky MT, Tanaka N, Abdulmalik O, Pavani G, Wang Y, Grupp SA, Chou ST, Ni H, Mui BL, Tam YK, Weissman D, Rivella S, Parhiz H. Breda L, et al. Science. 2023 Jul 28;381(6656):436-443. doi: 10.1126/science.ade6967. Epub 2023 Jul 27. Science. 2023. PMID: 37499029 Free PMC article.
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM. Russell S, et al. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Lancet. 2017. PMID: 28712537 Free PMC article. Clinical Trial.
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.
Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells.
Qin K, Huang P, Feng R, Keller CA, Peslak SA, Khandros E, Saari MS, Lan X, Mayuranathan T, Doerfler PA, Abdulmalik O, Giardine B, Chou ST, Shi J, Hardison RC, Weiss MJ, Blobel GA. Qin K, et al. Nat Genet. 2022 Jun;54(6):874-884. doi: 10.1038/s41588-022-01076-1. Epub 2022 May 26. Nat Genet. 2022. PMID: 35618846 Free PMC article.
HIC2 controls developmental hemoglobin switching by repressing BCL11A transcription.
Huang P, Peslak SA, Ren R, Khandros E, Qin K, Keller CA, Giardine B, Bell HW, Lan X, Sharma M, Horton JR, Abdulmalik O, Chou ST, Shi J, Crossley M, Hardison RC, Cheng X, Blobel GA. Huang P, et al. Nat Genet. 2022 Sep;54(9):1417-1426. doi: 10.1038/s41588-022-01152-6. Epub 2022 Aug 8. Nat Genet. 2022. PMID: 35941187 Free PMC article.
PIK3CA and CCM mutations fuel cavernomas through a cancer-like mechanism.
Ren AA, Snellings DA, Su YS, Hong CC, Castro M, Tang AT, Detter MR, Hobson N, Girard R, Romanos S, Lightle R, Moore T, Shenkar R, Benavides C, Beaman MM, Müller-Fielitz H, Chen M, Mericko P, Yang J, Sung DC, Lawton MT, Ruppert JM, Schwaninger M, Körbelin J, Potente M, Awad IA, Marchuk DA, Kahn ML. Ren AA, et al. Nature. 2021 Jun;594(7862):271-276. doi: 10.1038/s41586-021-03562-8. Epub 2021 Apr 28. Nature. 2021. PMID: 33910229 Free PMC article.
Cellular therapy: Immune-related complications.
Oved JH, Barrett DM, Teachey DT. Oved JH, et al. Immunol Rev. 2019 Jul;290(1):114-126. doi: 10.1111/imr.12768. Immunol Rev. 2019. PMID: 31355491 Free PMC article. Review.
Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy.
Chappell ME, Breda L, Tricoli L, Guerra A, Jarocha D, Castruccio Castracani C, Papp TE, Tanaka N, Hamilton N, Triebwasser MP, Ghiaccio V, Fedorky MT, Gollomp KL, Bochenek V, Roche AM, Everett JK, Cook EJ, Bushman FD, Teawtrakul N, Glentis S, Kattamis A, Mui BL, Tam YK, Weissman D, Abdulmalik O, Parhiz H, Rivella S. Chappell ME, et al. Blood. 2024 Oct 10;144(15):1633-1645. doi: 10.1182/blood.2023023349. Blood. 2024. PMID: 38949981
Hemophilia gene therapy comes of age.
George LA. George LA. Blood Adv. 2017 Dec 8;1(26):2591-2599. doi: 10.1182/bloodadvances.2017009878. eCollection 2017 Dec 12. Blood Adv. 2017. PMID: 29296912 Free PMC article. Review.
64 results