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

168 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
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. Among authors: roche am. 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.
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: roche am. 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.
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. Among authors: roche am. Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260. Blood. 2021. PMID: 33974038 Free PMC article. Clinical Trial.
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: roche am. 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.
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.
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. Among authors: roche am. 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.
Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome.
Labrosse R, Chu JI, Armant MA, Everett JK, Pellin D, Kareddy N, Frelinger AL, Henderson LA, O'Connell AE, Biswas A, Coenen-van der Spek J, Miggelbrink A, Fiorini C, Adhikari H, Berry CC, Cantu VA, Fong J, Jaroslavsky J, Karadeniz DF, Li QZ, Reddy S, Roche AM, Zhu C, Whangbo JS, Dansereau C, Mackinnon B, Morris E, Koo SM, London WB, Baris S, Ozen A, Karakoc-Aydiner E, Despotovic JM, Forbes Satter LR, Saitoh A, Aizawa Y, King A, Nguyen MAT, Vu VDU, Snapper SB, Galy A, Notarangelo LD, Bushman FD, Williams DA, Pai SY. Labrosse R, et al. Among authors: roche am. Blood. 2023 Oct 12;142(15):1281-1296. doi: 10.1182/blood.2022019117. Blood. 2023. PMID: 37478401 Free PMC article.
Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia.
Voit RA, Liao X, Caulier A, Antoszewski M, Cohen B, Armant M, Lu HY, Fleming TJ, Kamal E, Wahlster L, Roche AM, Everett JK, Petrichenko A, Huang MM, Clarke W, Myers KC, Forester C, Perez-Atayde A, Bushman FD, Pellin D, Shimamura A, Williams DA, Sankaran VG. Voit RA, et al. Among authors: roche am. Cell Stem Cell. 2025 Jan 2;32(1):38-52.e6. doi: 10.1016/j.stem.2024.10.012. Epub 2024 Nov 11. Cell Stem Cell. 2025. PMID: 39532107 Free PMC article.
168 results