These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

171 related articles for article (PubMed ID: 37188156)

  • 1. Specific correction of pyruvate kinase deficiency-causing point mutations by CRISPR/Cas9 and single-stranded oligodeoxynucleotides.
    Fañanas-Baquero S; Morín M; Fernández S; Ojeda-Perez I; Dessy-Rodriguez M; Giurgiu M; Bueren JA; Moreno-Pelayo MA; Segovia JC; Quintana-Bustamante O
    Front Genome Ed; 2023; 5():1104666. PubMed ID: 37188156
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
    Fañanas-Baquero S; Quintana-Bustamante O; Dever DP; Alberquilla O; Sanchez-Dominguez R; Camarena J; Ojeda-Perez I; Dessy-Rodriguez M; Turk R; Schubert MS; Lattanzi A; Xu L; Lopez-Lorenzo JL; Bianchi P; Bueren JA; Behlke MA; Porteus M; Segovia JC
    Mol Ther Methods Clin Dev; 2021 Sep; 22():237-248. PubMed ID: 34485608
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA.
    Quintana-Bustamante O; Fañanas-Baquero S; Orman I; Torres R; Duchateau P; Poirot L; Gouble A; Bueren JA; Segovia JC
    PLoS One; 2019; 14(10):e0223775. PubMed ID: 31618280
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Challenges in Gene Therapy for Somatic Reverted Mosaicism in X-Linked Combined Immunodeficiency by CRISPR/Cas9 and Prime Editing.
    Hou Y; Ureña-Bailén G; Mohammadian Gol T; Gratz PG; Gratz HP; Roig-Merino A; Antony JS; Lamsfus-Calle A; Daniel-Moreno A; Handgretinger R; Mezger M
    Genes (Basel); 2022 Dec; 13(12):. PubMed ID: 36553615
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9.
    Paquet D; Kwart D; Chen A; Sproul A; Jacob S; Teo S; Olsen KM; Gregg A; Noggle S; Tessier-Lavigne M
    Nature; 2016 May; 533(7601):125-9. PubMed ID: 27120160
    [TBL] [Abstract][Full Text] [Related]  

  • 6. A Family Affected by a Life-Threatening Erythrocyte Defect Caused by Pyruvate Kinase Deficiency With Normal Iron Status: A Case Report.
    Maciak K; Adamowicz-Salach A; Poznanski J; Gora M; Fronk J; Burzynska B
    Front Genet; 2020; 11():560248. PubMed ID: 33193643
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Mutation-Specific Guide RNA for Compound Heterozygous Porphyria On-target Scarless Correction by CRISPR/Cas9 in Stem Cells.
    Prat F; Toutain J; Boutin J; Amintas S; Cullot G; Lalanne M; Lamrissi-Garcia I; Moranvillier I; Richard E; Blouin JM; Dabernat S; Moreau-Gaudry F; Bedel A
    Stem Cell Reports; 2020 Sep; 15(3):677-693. PubMed ID: 32795423
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells.
    Garate Z; Quintana-Bustamante O; Crane AM; Olivier E; Poirot L; Galetto R; Kosinski P; Hill C; Kung C; Agirre X; Orman I; Cerrato L; Alberquilla O; Rodriguez-Fornes F; Fusaki N; Garcia-Sanchez F; Maia TM; Ribeiro ML; Sevilla J; Prosper F; Jin S; Mountford J; Guenechea G; Gouble A; Bueren JA; Davis BR; Segovia JC
    Stem Cell Reports; 2015 Dec; 5(6):1053-1066. PubMed ID: 26549847
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Safe and Efficient Gene Therapy for Pyruvate Kinase Deficiency.
    Garcia-Gomez M; Calabria A; Garcia-Bravo M; Benedicenti F; Kosinski P; López-Manzaneda S; Hill C; Del Mar Mañu-Pereira M; Martín MA; Orman I; Vives-Corrons JL; Kung C; Schambach A; Jin S; Bueren JA; Montini E; Navarro S; Segovia JC
    Mol Ther; 2016 Aug; 24(7):1187-98. PubMed ID: 27138040
    [TBL] [Abstract][Full Text] [Related]  

  • 10. CRISPR-targeted genome editing of human induced pluripotent stem cell-derived hepatocytes for the treatment of Wilson's disease.
    Wei R; Yang J; Cheng CW; Ho WI; Li N; Hu Y; Hong X; Fu J; Yang B; Liu Y; Jiang L; Lai WH; Au KW; Tsang WL; Tse YL; Ng KM; Esteban MA; Tse HF
    JHEP Rep; 2022 Jan; 4(1):100389. PubMed ID: 34877514
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Ligase IV inhibitor SCR7 enhances gene editing directed by CRISPR-Cas9 and ssODN in human cancer cells.
    Hu Z; Shi Z; Guo X; Jiang B; Wang G; Luo D; Chen Y; Zhu YS
    Cell Biosci; 2018; 8():12. PubMed ID: 29468011
    [TBL] [Abstract][Full Text] [Related]  

  • 12. A novel and a previously described compound heterozygous PKLR gene mutations causing pyruvate kinase deficiency in a Chinese child.
    Li H; Gu P; Yao RE; Wang J; Fu Q; Wang J
    Fetal Pediatr Pathol; 2014 Jun; 33(3):182-90. PubMed ID: 24601847
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Optimizing CRISPR/Cas9 technology for precise correction of the Fgfr3-G374R mutation in achondroplasia in mice.
    Miao K; Zhang X; Su SM; Zeng J; Huang Z; Chan UI; Xu X; Deng CX
    J Biol Chem; 2019 Jan; 294(4):1142-1151. PubMed ID: 30487289
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Gene correction of HBB mutations in CD34
    Antony JS; Latifi N; Haque AKMA; Lamsfus-Calle A; Daniel-Moreno A; Graeter S; Baskaran P; Weinmann P; Mezger M; Handgretinger R; Kormann MSD
    Mol Cell Pediatr; 2018 Nov; 5(1):9. PubMed ID: 30430274
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
    Navarro S; Quintana-Bustamante O; Sanchez-Dominguez R; Lopez-Manzaneda S; Ojeda-Perez I; Garcia-Torralba A; Alberquilla O; Law K; Beard BC; Bastone A; Rothe M; Villanueva M; Ramirez JC; Fañanas-Baquero S; Nieto-Romero V; Molinos-Vicente A; Gutierrez S; Nicoletti E; García-Bravo M; Bueren JA; Schwartz JD; Segovia JC
    Mol Ther Methods Clin Dev; 2021 Sep; 22():350-359. PubMed ID: 34514027
    [TBL] [Abstract][Full Text] [Related]  

  • 16. The most common disease-causing mutation of factor XIII deficiency is corrected by CRISPR/CAS9 gene editing system.
    Dorgalaleh A; Kiani J; Zaker F; Safa M
    Blood Coagul Fibrinolysis; 2022 Apr; 33(3):153-158. PubMed ID: 35221320
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Efficient gene correction of an aberrant splice site in β-thalassaemia iPSCs by CRISPR/Cas9 and single-strand oligodeoxynucleotides.
    Xiong Z; Xie Y; Yang Y; Xue Y; Wang D; Lin S; Chen D; Lu D; He L; Song B; Yang Y; Sun X
    J Cell Mol Med; 2019 Dec; 23(12):8046-8057. PubMed ID: 31631510
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Efficient and allele-specific genome editing of disease loci in human iPSCs.
    Smith C; Abalde-Atristain L; He C; Brodsky BR; Braunstein EM; Chaudhari P; Jang YY; Cheng L; Ye Z
    Mol Ther; 2015 Mar; 23(3):570-7. PubMed ID: 25418680
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Gene Editing for Inherited Red Blood Cell Diseases.
    Quintana-Bustamante O; Fañanas-Baquero S; Dessy-Rodriguez M; Ojeda-Pérez I; Segovia JC
    Front Physiol; 2022; 13():848261. PubMed ID: 35418876
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Rescue of pyruvate kinase deficiency in mice by gene therapy using the human isoenzyme.
    Meza NW; Alonso-Ferrero ME; Navarro S; Quintana-Bustamante O; Valeri A; Garcia-Gomez M; Bueren JA; Bautista JM; Segovia JC
    Mol Ther; 2009 Dec; 17(12):2000-9. PubMed ID: 19755962
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.