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Table representation of search results timeline featuring number of search results per year.
Year | Number of Results |
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2016 | 1 |
2021 | 2 |
2024 | 0 |
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Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair.
Gene Ther. 2021 Jun;28(6):373-390. doi: 10.1038/s41434-021-00251-z. Epub 2021 Mar 12.
Gene Ther. 2021.
PMID: 33712802
Free PMC article.
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Schejtman A, Vetharoy W, Choi U, Rivat C, Theobald N, Piras G, Leon-Rico D, Buckland K, Armenteros-Monterroso E, Benedetti S, Ashworth MT, Rothe M, Schambach A, Gaspar HB, Kang EM, Malech HL, Thrasher AJ, Santilli G.
Schejtman A, et al.
Hum Gene Ther. 2021 Sep;32(17-18):949-958. doi: 10.1089/hum.2020.276. Epub 2021 May 6.
Hum Gene Ther. 2021.
PMID: 33740872
Free PMC article.
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Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease.
De Ravin SS, Reik A, Liu PQ, Li L, Wu X, Su L, Raley C, Theobald N, Choi U, Song AH, Chan A, Pearl JR, Paschon DE, Lee J, Newcombe H, Koontz S, Sweeney C, Shivak DA, Zarember KA, Peshwa MV, Gregory PD, Urnov FD, Malech HL.
De Ravin SS, et al.
Nat Biotechnol. 2016 Apr;34(4):424-9. doi: 10.1038/nbt.3513. Epub 2016 Mar 7.
Nat Biotechnol. 2016.
PMID: 26950749
Free PMC article.
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