378 related articles for article (PubMed ID: 31402115)
1. Targeted gene therapy in human-induced pluripotent stem cells from a patient with primary hyperoxaluria type 1 using CRISPR/Cas9 technology.
Estève J; Blouin JM; Lalanne M; Azzi-Martin L; Dubus P; Bidet A; Harambat J; Llanas B; Moranvillier I; Bedel A; Moreau-Gaudry F; Richard E
Biochem Biophys Res Commun; 2019 Oct; 517(4):677-683. PubMed ID: 31402115
[TBL] [Abstract][Full Text] [Related]
2. Generation of induced pluripotent stem cells-derived hepatocyte-like cells for ex vivo gene therapy of primary hyperoxaluria type 1.
Estève J; Blouin JM; Lalanne M; Azzi-Martin L; Dubus P; Bidet A; Harambat J; Llanas B; Moranvillier I; Bedel A; Moreau-Gaudry F; Richard E
Stem Cell Res; 2019 Jul; 38():101467. PubMed ID: 31151050
[TBL] [Abstract][Full Text] [Related]
3. Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system.
Lyu C; Shen J; Wang R; Gu H; Zhang J; Xue F; Liu X; Liu W; Fu R; Zhang L; Li H; Zhang X; Cheng T; Yang R; Zhang L
Stem Cell Res Ther; 2018 Apr; 9(1):92. PubMed ID: 29625575
[TBL] [Abstract][Full Text] [Related]
4. Generation of a Primary Hyperoxaluria Type 1 Disease Model Via CRISPR/Cas9 System in Rats.
Zheng R; Fang X; He L; Shao Y; Guo N; Wang L; Liu M; Li D; Geng H
Curr Mol Med; 2018; 18(7):436-447. PubMed ID: 30539697
[TBL] [Abstract][Full Text] [Related]
5. CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I.
Zabaleta N; Barberia M; Martin-Higueras C; Zapata-Linares N; Betancor I; Rodriguez S; Martinez-Turrillas R; Torella L; Vales A; Olagüe C; Vilas-Zornoza A; Castro-Labrador L; Lara-Astiaso D; Prosper F; Salido E; Gonzalez-Aseguinolaza G; Rodriguez-Madoz JR
Nat Commun; 2018 Dec; 9(1):5454. PubMed ID: 30575740
[TBL] [Abstract][Full Text] [Related]
6. Generation and characterization of a novel rat model of primary hyperoxaluria type 1 with a nonsense mutation in alanine-glyoxylate aminotransferase gene.
Li Y; Zheng R; Xu G; Huang Y; Li Y; Li D; Geng H
Am J Physiol Renal Physiol; 2021 Mar; 320(3):F475-F484. PubMed ID: 33491567
[TBL] [Abstract][Full Text] [Related]
7. Differential Transgene Silencing of Myeloid-Specific Promoters in the
Klatt D; Cheng E; Hoffmann D; Santilli G; Thrasher AJ; Brendel C; Schambach A
Hum Gene Ther; 2020 Feb; 31(3-4):199-210. PubMed ID: 31773990
[TBL] [Abstract][Full Text] [Related]
8. Correction of hyperoxaluria by liver repopulation with hepatocytes in a mouse model of primary hyperoxaluria type-1.
Jiang J; Salido EC; Guha C; Wang X; Moitra R; Liu L; Roy-Chowdhury J; Roy-Chowdhury N
Transplantation; 2008 May; 85(9):1253-60. PubMed ID: 18475180
[TBL] [Abstract][Full Text] [Related]
9. Low-density lipoprotein receptor-deficient hepatocytes differentiated from induced pluripotent stem cells allow familial hypercholesterolemia modeling, CRISPR/Cas-mediated genetic correction, and productive hepatitis C virus infection.
Caron J; Pène V; Tolosa L; Villaret M; Luce E; Fourrier A; Heslan JM; Saheb S; Bruckert E; Gómez-Lechón MJ; Nguyen TH; Rosenberg AR; Weber A; Dubart-Kupperschmitt A
Stem Cell Res Ther; 2019 Jul; 10(1):221. PubMed ID: 31358055
[TBL] [Abstract][Full Text] [Related]
10. CRISPR/Cas9-mediated metabolic pathway reprogramming in a novel humanized rat model ameliorates primary hyperoxaluria type 1.
Zheng R; Li Y; Wang L; Fang X; Zhang J; He L; Yang L; Li D; Geng H
Kidney Int; 2020 Oct; 98(4):947-957. PubMed ID: 32464217
[TBL] [Abstract][Full Text] [Related]
11. CRISPR/Cas9-mediated gene correction in hemophilia B patient-derived iPSCs.
Morishige S; Mizuno S; Ozawa H; Nakamura T; Mazahery A; Nomura K; Seki R; Mouri F; Osaki K; Yamamura K; Okamura T; Nagafuji K
Int J Hematol; 2020 Feb; 111(2):225-233. PubMed ID: 31664646
[TBL] [Abstract][Full Text] [Related]
12. Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1.
Kukreja A; Lasaro M; Cobaugh C; Forbes C; Tang JP; Gao X; Martin-Higueras C; Pey AL; Salido E; Sobolov S; Subramanian RR
Nucleic Acid Ther; 2019 Apr; 29(2):104-113. PubMed ID: 30676254
[TBL] [Abstract][Full Text] [Related]
13. Generation of an induced pluripotent stem cell line (CIMAi001-A) from a compound heterozygous Primary Hyperoxaluria Type I (PH1) patient carrying p.G170R and p.R122* mutations in the AGXT gene.
Martinez-Turrillas R; Rodriguez-Diaz S; Rodriguez-Marquez P; Martin-Mallo A; Salido E; Beck BB; Prosper F; Rodriguez-Madoz JR
Stem Cell Res; 2019 Dec; 41():101626. PubMed ID: 31715429
[TBL] [Abstract][Full Text] [Related]
14. In vivo base editing rescues primary hyperoxaluria type 1 in rats.
Chen Z; Zhang D; Zheng R; Yang L; Huo Y; Zhang D; Fang X; Li Y; Xu G; Li D; Geng H
Kidney Int; 2024 Mar; 105(3):496-507. PubMed ID: 38142039
[TBL] [Abstract][Full Text] [Related]
15. Restored glyoxylate metabolism after AGXT gene correction and direct reprogramming of primary hyperoxaluria type 1 fibroblasts.
Nieto-Romero V; García-Torralba A; Molinos-Vicente A; Moya FJ; Rodríguez-Perales S; García-Escudero R; Salido E; Segovia JC; García-Bravo M
iScience; 2024 Apr; 27(4):109530. PubMed ID: 38577102
[TBL] [Abstract][Full Text] [Related]
16. Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1.
Castello R; Borzone R; D'Aria S; Annunziata P; Piccolo P; Brunetti-Pierri N
Gene Ther; 2016 Feb; 23(2):129-34. PubMed ID: 26609667
[TBL] [Abstract][Full Text] [Related]
17. Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer.
Salido E; Rodriguez-Pena M; Santana A; Beattie SG; Petry H; Torres A
Mol Ther; 2011 May; 19(5):870-5. PubMed ID: 21119625
[TBL] [Abstract][Full Text] [Related]
18. CRISPR-Cas9-Based Genome Editing of Human Induced Pluripotent Stem Cells.
Giacalone JC; Sharma TP; Burnight ER; Fingert JF; Mullins RF; Stone EM; Tucker BA
Curr Protoc Stem Cell Biol; 2018 Feb; 44():5B.7.1-5B.7.22. PubMed ID: 29512106
[TBL] [Abstract][Full Text] [Related]
19. Multiplex gene editing reduces oxalate production in primary hyperoxaluria type 1.
Zheng R; Zhang DX; Shao YJ; Fang XL; Yang L; Huo YN; Li DL; Geng HQ
Zool Res; 2023 Nov; 44(6):993-1002. PubMed ID: 37759334
[TBL] [Abstract][Full Text] [Related]
20. Curative Ex Vivo Hepatocyte-Directed Gene Editing in a Mouse Model of Hereditary Tyrosinemia Type 1.
VanLith C; Guthman R; Nicolas CT; Allen K; Du Z; Joo DJ; Nyberg SL; Lillegard JB; Hickey RD
Hum Gene Ther; 2018 Nov; 29(11):1315-1326. PubMed ID: 29764210
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]