BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

219 related articles for article (PubMed ID: 37080708)

  • 1. Gene editing for sickle cell disease and transfusion dependent thalassemias- A cure within reach.
    Eckrich MJ; Frangoul H
    Semin Hematol; 2023 Jan; 60(1):3-9. PubMed ID: 37080708
    [TBL] [Abstract][Full Text] [Related]  

  • 2. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
    Frangoul H; Altshuler D; Cappellini MD; Chen YS; Domm J; Eustace BK; Foell J; de la Fuente J; Grupp S; Handgretinger R; Ho TW; Kattamis A; Kernytsky A; Lekstrom-Himes J; Li AM; Locatelli F; Mapara MY; de Montalembert M; Rondelli D; Sharma A; Sheth S; Soni S; Steinberg MH; Wall D; Yen A; Corbacioglu S
    N Engl J Med; 2021 Jan; 384(3):252-260. PubMed ID: 33283989
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies.
    Paschoudi K; Yannaki E; Psatha N
    Int J Mol Sci; 2023 May; 24(11):. PubMed ID: 37298481
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia.
    Ye L; Wang J; Tan Y; Beyer AI; Xie F; Muench MO; Kan YW
    Proc Natl Acad Sci U S A; 2016 Sep; 113(38):10661-5. PubMed ID: 27601644
    [TBL] [Abstract][Full Text] [Related]  

  • 5. CRISPR/Cas9 gene editing for curing sickle cell disease.
    Park SH; Bao G
    Transfus Apher Sci; 2021 Feb; 60(1):103060. PubMed ID: 33455878
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Gene therapy for sickle cell disease: An update.
    Demirci S; Uchida N; Tisdale JF
    Cytotherapy; 2018 Jul; 20(7):899-910. PubMed ID: 29859773
    [TBL] [Abstract][Full Text] [Related]  

  • 7. A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.
    Traxler EA; Yao Y; Wang YD; Woodard KJ; Kurita R; Nakamura Y; Hughes JR; Hardison RC; Blobel GA; Li C; Weiss MJ
    Nat Med; 2016 Sep; 22(9):987-90. PubMed ID: 27525524
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype.
    Weber L; Frati G; Felix T; Hardouin G; Casini A; Wollenschlaeger C; Meneghini V; Masson C; De Cian A; Chalumeau A; Mavilio F; Amendola M; Andre-Schmutz I; Cereseto A; El Nemer W; Concordet JP; Giovannangeli C; Cavazzana M; Miccio A
    Sci Adv; 2020 Feb; 6(7):. PubMed ID: 32917636
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease.
    Germino-Watnick P; Hinds M; Le A; Chu R; Liu X; Uchida N
    Cells; 2022 Jun; 11(11):. PubMed ID: 35681538
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice.
    Sii-Felice K; Giorgi M; Leboulch P; Payen E
    Exp Hematol; 2018 Aug; 64():12-32. PubMed ID: 29807062
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies.
    Magrin E; Miccio A; Cavazzana M
    Blood; 2019 Oct; 134(15):1203-1213. PubMed ID: 31467062
    [TBL] [Abstract][Full Text] [Related]  

  • 12. New therapeutic targets in transfusion-dependent and -independent thalassemia.
    Cappellini MD; Motta I
    Hematology Am Soc Hematol Educ Program; 2017 Dec; 2017(1):278-283. PubMed ID: 29222267
    [TBL] [Abstract][Full Text] [Related]  

  • 13. CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia.
    Zeng S; Lei S; Qu C; Wang Y; Teng S; Huang P
    Hum Genet; 2023 Dec; 142(12):1677-1703. PubMed ID: 37878144
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.
    Wilkinson AC; Dever DP; Baik R; Camarena J; Hsu I; Charlesworth CT; Morita C; Nakauchi H; Porteus MH
    Nat Commun; 2021 Jan; 12(1):686. PubMed ID: 33514718
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders.
    Lohani N; Bhargava N; Munshi A; Ramalingam S
    J Cell Physiol; 2018 Jun; 233(6):4563-4577. PubMed ID: 29159826
    [TBL] [Abstract][Full Text] [Related]  

  • 16. The Novel Role of the B-Cell Lymphoma/Leukemia 11A (BCL11A) Gene in β-Thalassaemia Treatment.
    Mahmoud Ahmed NH; Lai MI
    Cardiovasc Hematol Disord Drug Targets; 2023; 22(4):226-236. PubMed ID: 36734897
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.
    Ramadier S; Chalumeau A; Felix T; Othman N; Aknoun S; Casini A; Maule G; Masson C; De Cian A; Frati G; Brusson M; Concordet JP; Cavazzana M; Cereseto A; El Nemer W; Amendola M; Wattellier B; Meneghini V; Miccio A
    Mol Ther; 2022 Jan; 30(1):145-163. PubMed ID: 34418541
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin.
    Ravi NS; Wienert B; Wyman SK; Bell HW; George A; Mahalingam G; Vu JT; Prasad K; Bandlamudi BP; Devaraju N; Rajendiran V; Syedbasha N; Pai AA; Nakamura Y; Kurita R; Narayanasamy M; Balasubramanian P; Thangavel S; Marepally S; Velayudhan SR; Srivastava A; DeWitt MA; Crossley M; Corn JE; Mohankumar KM
    Elife; 2022 Feb; 11():. PubMed ID: 35147495
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia.
    Thuret I; Ruggeri A; Angelucci E; Chabannon C
    Stem Cells Transl Med; 2022 Apr; 11(4):407-414. PubMed ID: 35267028
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Genome editing approaches to β-hemoglobinopathies.
    Brusson M; Miccio A
    Prog Mol Biol Transl Sci; 2021; 182():153-183. PubMed ID: 34175041
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 11.