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 NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

347 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: roncarolo mg. Nat Commun. 2019 Apr 9;10(1):1634. doi: 10.1038/s41467-019-09614-y. Nat Commun. 2019. PMID: 30967552 Free PMC article.
Role of human forkhead box P3 in early thymic maturation and peripheral T-cell homeostasis.
Santoni de Sio FR, Passerini L, Restelli S, Valente MM, Pramov A, Maccari ME, Sanvito F, Roncarolo MG, Porteus M, Bacchetta R. Santoni de Sio FR, et al. Among authors: roncarolo mg. J Allergy Clin Immunol. 2018 Dec;142(6):1909-1921.e9. doi: 10.1016/j.jaci.2018.03.015. Epub 2018 Apr 27. J Allergy Clin Immunol. 2018. PMID: 29705245
Reprogramming human T cell function and specificity with non-viral genome targeting.
Roth TL, Puig-Saus C, Yu R, Shifrut E, Carnevale J, Li PJ, Hiatt J, Saco J, Krystofinski P, Li H, Tobin V, Nguyen DN, Lee MR, Putnam AL, Ferris AL, Chen JW, Schickel JN, Pellerin L, Carmody D, Alkorta-Aranburu G, Del Gaudio D, Matsumoto H, Morell M, Mao Y, Cho M, Quadros RM, Gurumurthy CB, Smith B, Haugwitz M, Hughes SH, Weissman JS, Schumann K, Esensten JH, May AP, Ashworth A, Kupfer GM, Greeley SAW, Bacchetta R, Meffre E, Roncarolo MG, Romberg N, Herold KC, Ribas A, Leonetti MD, Marson A. Roth TL, et al. Among authors: roncarolo mg. Nature. 2018 Jul;559(7714):405-409. doi: 10.1038/s41586-018-0326-5. Epub 2018 Jul 11. Nature. 2018. PMID: 29995861 Free PMC article.
Molecular and functional heterogeneity of IL-10-producing CD4+ T cells.
Brockmann L, Soukou S, Steglich B, Czarnewski P, Zhao L, Wende S, Bedke T, Ergen C, Manthey C, Agalioti T, Geffken M, Seiz O, Parigi SM, Sorini C, Geginat J, Fujio K, Jacobs T, Roesch T, Izbicki JR, Lohse AW, Flavell RA, Krebs C, Gustafsson JA, Antonson P, Roncarolo MG, Villablanca EJ, Gagliani N, Huber S. Brockmann L, et al. Among authors: roncarolo mg. Nat Commun. 2018 Dec 21;9(1):5457. doi: 10.1038/s41467-018-07581-4. Nat Commun. 2018. PMID: 30575716 Free PMC article.
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: roncarolo mg. Nat Commun. 2019 Apr 26;10(1):2021. doi: 10.1038/s41467-019-10080-9. Nat Commun. 2019. PMID: 31028274 Free PMC article.
Gene therapy for primary immunodeficiency.
Booth C, Romano R, Roncarolo MG, Thrasher AJ. Booth C, et al. Among authors: roncarolo mg. Hum Mol Genet. 2019 Oct 1;28(R1):R15-R23. doi: 10.1093/hmg/ddz170. Hum Mol Genet. 2019. PMID: 31297531 Review.
Author Correction: Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH. Pavel-Dinu M, et al. Among authors: roncarolo mg. Nat Commun. 2019 Dec 4;10(1):5624. doi: 10.1038/s41467-019-13620-5. Nat Commun. 2019. PMID: 31796738 Free PMC article.
Genome editing of donor-derived T-cells to generate allogenic chimeric antigen receptor-modified T cells: Optimizing αβ T cell-depleted haploidentical hematopoietic stem cell transplantation.
Wiebking V, Lee CM, Mostrel N, Lahiri P, Bak R, Bao G, Roncarolo MG, Bertaina A, Porteus MH. Wiebking V, et al. Among authors: roncarolo mg. Haematologica. 2021 Mar 1;106(3):847-858. doi: 10.3324/haematol.2019.233882. Haematologica. 2021. PMID: 32241852 Free PMC article.
CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells.
Goodwin M, Lee E, Lakshmanan U, Shipp S, Froessl L, Barzaghi F, Passerini L, Narula M, Sheikali A, Lee CM, Bao G, Bauer CS, Miller HK, Garcia-Lloret M, Butte MJ, Bertaina A, Shah A, Pavel-Dinu M, Hendel A, Porteus M, Roncarolo MG, Bacchetta R. Goodwin M, et al. Among authors: roncarolo mg. Sci Adv. 2020 May 6;6(19):eaaz0571. doi: 10.1126/sciadv.aaz0571. eCollection 2020 May. Sci Adv. 2020. PMID: 32494707 Free PMC article.
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Among authors: roncarolo mg. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
347 results