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Page 1
Lentiviral gene therapy for X-linked chronic granulomatous disease.
Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, Thrasher AJ; Net4CGD consortium. Kohn DB, et al. Among authors: everett jk. Nat Med. 2020 Feb;26(2):200-206. doi: 10.1038/s41591-019-0735-5. Epub 2020 Jan 27. Nat Med. 2020. PMID: 31988463 Free PMC article. Clinical Trial.
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.
Magnani A, Semeraro M, Adam F, Booth C, Dupré L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Magnani A, et al. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24. Nat Med. 2022. PMID: 35075289 Free PMC article.
Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. Reinhardt B, et al. Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260. Blood. 2021. PMID: 33974038 Free PMC article. Clinical Trial.
Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.
Magnani A, Semeraro M, Adam F, Booth C, Dupré L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Magnani A, et al. Nat Med. 2022 Oct;28(10):2217. doi: 10.1038/s41591-022-01985-y. Nat Med. 2022. PMID: 35945284 Free PMC article. No abstract available.
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency.
Clarke EL, Connell AJ, Six E, Kadry NA, Abbas AA, Hwang Y, Everett JK, Hofstaedter CE, Marsh R, Armant M, Kelsen J, Notarangelo LD, Collman RG, Hacein-Bey-Abina S, Kohn DB, Cavazzana M, Fischer A, Williams DA, Pai SY, Bushman FD. Clarke EL, et al. Among authors: everett jk. Genome Med. 2018 Sep 28;10(1):70. doi: 10.1186/s13073-018-0580-z. Genome Med. 2018. PMID: 30261899 Free PMC article.
Lentiviral globin gene therapy with reduced-intensity conditioning in adults with β-thalassemia: a phase 1 trial.
Boulad F, Maggio A, Wang X, Moi P, Acuto S, Kogel F, Takpradit C, Prockop S, Mansilla-Soto J, Cabriolu A, Odak A, Qu J, Thummar K, Du F, Shen L, Raso S, Barone R, Di Maggio R, Pitrolo L, Giambona A, Mingoia M, Everett JK, Hokama P, Roche AM, Cantu VA, Adhikari H, Reddy S, Bouhassira E, Mohandas N, Bushman FD, Rivière I, Sadelain M. Boulad F, et al. Among authors: everett jk. Nat Med. 2022 Jan;28(1):63-70. doi: 10.1038/s41591-021-01554-9. Epub 2022 Jan 3. Nat Med. 2022. PMID: 34980909 Free PMC article. Clinical Trial.
Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs.
Six E, Guilloux A, Denis A, Lecoules A, Magnani A, Vilette R, Male F, Cagnard N, Delville M, Magrin E, Caccavelli L, Roudaut C, Plantier C, Sobrino S, Gregg J, Nobles CL, Everett JK, Hacein-Bey-Abina S, Galy A, Fischer A, Thrasher AJ, André I, Cavazzana M, Bushman FD. Six E, et al. Among authors: everett jk. Blood. 2020 Apr 9;135(15):1219-1231. doi: 10.1182/blood.2019002350. Blood. 2020. PMID: 32040546 Free PMC article. Clinical Trial.
A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
Nguyen GN, Everett JK, Kafle S, Roche AM, Raymond HE, Leiby J, Wood C, Assenmacher CA, Merricks EP, Long CT, Kazazian HH, Nichols TC, Bushman FD, Sabatino DE. Nguyen GN, et al. Among authors: everett jk. Nat Biotechnol. 2021 Jan;39(1):47-55. doi: 10.1038/s41587-020-0741-7. Epub 2020 Nov 16. Nat Biotechnol. 2021. PMID: 33199875 Free PMC article.
PSMA-targeting TGFβ-insensitive armored CAR T cells in metastatic castration-resistant prostate cancer: a phase 1 trial.
Narayan V, Barber-Rotenberg JS, Jung IY, Lacey SF, Rech AJ, Davis MM, Hwang WT, Lal P, Carpenter EL, Maude SL, Plesa G, Vapiwala N, Chew A, Moniak M, Sebro RA, Farwell MD, Marshall A, Gilmore J, Lledo L, Dengel K, Church SE, Hether TD, Xu J, Gohil M, Buckingham TH, Yee SS, Gonzalez VE, Kulikovskaya I, Chen F, Tian L, Tien K, Gladney W, Nobles CL, Raymond HE; Prostate Cancer Cellular Therapy Program Investigators; Hexner EO, Siegel DL, Bushman FD, June CH, Fraietta JA, Haas NB. Narayan V, et al. Nat Med. 2022 Apr;28(4):724-734. doi: 10.1038/s41591-022-01726-1. Epub 2022 Mar 21. Nat Med. 2022. PMID: 35314843 Free PMC article. Clinical Trial.
73 results