BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

133 related articles for article (PubMed ID: 38691622)

  • 1. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
    Consiglieri G; Tucci F; De Pellegrin M; Guerrini B; Cattoni A; Risca G; Scarparo S; Sarzana M; Pontesilli S; Mellone R; Gasperini S; Galimberti S; Silvani P; Filisetti C; Darin S; Forni G; Miglietta S; Santi L; Facchini M; Corti A; Fumagalli F; Cicalese MP; Calbi V; Migliavacca M; Barzaghi F; Ferrua F; Gallo V; Recupero S; Canarutto D; Doglio M; Tedesco L; Volpi N; Rovelli A; la Marca G; Valsecchi MG; Zancan S; Ciceri F; Naldini L; Baldoli C; Parini R; Gentner B; Aiuti A; Bernardo ME
    Sci Transl Med; 2024 May; 16(745):eadi8214. PubMed ID: 38691622
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
    Gentner B; Tucci F; Galimberti S; Fumagalli F; De Pellegrin M; Silvani P; Camesasca C; Pontesilli S; Darin S; Ciotti F; Sarzana M; Consiglieri G; Filisetti C; Forni G; Passerini L; Tomasoni D; Cesana D; Calabria A; Spinozzi G; Cicalese MP; Calbi V; Migliavacca M; Barzaghi F; Ferrua F; Gallo V; Miglietta S; Zonari E; Cheruku PS; Forni C; Facchini M; Corti A; Gabaldo M; Zancan S; Gasperini S; Rovelli A; Boelens JJ; Jones SA; Wynn R; Baldoli C; Montini E; Gregori S; Ciceri F; Valsecchi MG; la Marca G; Parini R; Naldini L; Aiuti A; Bernardo ME;
    N Engl J Med; 2021 Nov; 385(21):1929-1940. PubMed ID: 34788506
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Musculoskeletal manifestations in mucopolysaccharidosis type I (Hurler syndrome) following hematopoietic stem cell transplantation.
    Schmidt M; Breyer S; Löbel U; Yarar S; Stücker R; Ullrich K; Müller I; Muschol N
    Orphanet J Rare Dis; 2016 Jul; 11(1):93. PubMed ID: 27392569
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Long term disease burden post-transplantation: three decades of observations in 25 Hurler patients successfully treated with hematopoietic stem cell transplantation (HSCT).
    Guffon N; Pettazzoni M; Pangaud N; Garin C; Lina-Granade G; Plault C; Mottolese C; Froissart R; Fouilhoux A
    Orphanet J Rare Dis; 2021 Jan; 16(1):60. PubMed ID: 33517895
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Progression of Hip Dysplasia in Mucopolysaccharidosis Type I Hurler After Successful Hematopoietic Stem Cell Transplantation.
    Langereis EJ; den Os MM; Breen C; Jones SA; Knaven OC; Mercer J; Miller WP; Kelly PM; Kennedy J; Ketterl TG; O'Meara A; Orchard PJ; Lund TC; van Rijn RR; Sakkers RJ; White KK; Wijburg FA
    J Bone Joint Surg Am; 2016 Mar; 98(5):386-95. PubMed ID: 26935461
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.
    Ferrua F; Cicalese MP; Galimberti S; Giannelli S; Dionisio F; Barzaghi F; Migliavacca M; Bernardo ME; Calbi V; Assanelli AA; Facchini M; Fossati C; Albertazzi E; Scaramuzza S; Brigida I; Scala S; Basso-Ricci L; Pajno R; Casiraghi M; Canarutto D; Salerio FA; Albert MH; Bartoli A; Wolf HM; Fiori R; Silvani P; Gattillo S; Villa A; Biasco L; Dott C; Culme-Seymour EJ; van Rossem K; Atkinson G; Valsecchi MG; Roncarolo MG; Ciceri F; Naldini L; Aiuti A
    Lancet Haematol; 2019 May; 6(5):e239-e253. PubMed ID: 30981783
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I.
    Watson HA; Holley RJ; Langford-Smith KJ; Wilkinson FL; van Kuppevelt TH; Wynn RF; Wraith JE; Merry CL; Bigger BW
    J Biol Chem; 2014 Dec; 289(52):36194-203. PubMed ID: 25359774
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Analysis of factors affecting development of carpal tunnel syndrome in patients with Hurler syndrome after hematopoietic cell transplantation.
    Khanna G; Van Heest AE; Agel J; Bjoraker K; Grewal S; Abel S; Krivit W; Peters C; Orchard PJ
    Bone Marrow Transplant; 2007 Mar; 39(6):331-4. PubMed ID: 17277793
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Outcome in six children with mucopolysaccharidosis type IH, Hurler syndrome, after haematopoietic stem cell transplantation (HSCT).
    Malm G; Gustafsson B; Berglund G; Lindström M; Naess K; Borgström B; von Döbeln U; Ringdén O
    Acta Paediatr; 2008 Aug; 97(8):1108-12. PubMed ID: 18452566
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Long-term outcomes of adaptive functions for children with mucopolysaccharidosis I (Hurler syndrome) treated with hematopoietic stem cell transplantation.
    Bjoraker KJ; Delaney K; Peters C; Krivit W; Shapiro EG
    J Dev Behav Pediatr; 2006 Aug; 27(4):290-6. PubMed ID: 16906003
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
    Visigalli I; Delai S; Politi LS; Di Domenico C; Cerri F; Mrak E; D'Isa R; Ungaro D; Stok M; Sanvito F; Mariani E; Staszewsky L; Godi C; Russo I; Cecere F; Del Carro U; Rubinacci A; Brambilla R; Quattrini A; Di Natale P; Ponder K; Naldini L; Biffi A
    Blood; 2010 Dec; 116(24):5130-9. PubMed ID: 20847202
    [TBL] [Abstract][Full Text] [Related]  

  • 12. The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome).
    Miebach E; Church H; Cooper A; Mercer J; Tylee K; Wynn RF; Wraith JE
    J Inherit Metab Dis; 2011 Jun; 34(3):755-61. PubMed ID: 21416193
    [TBL] [Abstract][Full Text] [Related]  

  • 13. The evolution of pulmonary function in childhood onset Mucopolysaccharidosis type I.
    Broomfield A; Sims J; Mercer J; Hensman P; Ghosh A; Tylee K; Stepien KM; Oldham A; Prathivadi Bhayankaram N; Wynn R; Wright NB; Jones SA; Wilkinson S
    Mol Genet Metab; 2021 Feb; 132(2):94-99. PubMed ID: 32713717
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation.
    Polgreen LE; Tolar J; Plog M; Himes JH; Orchard PJ; Whitley CB; Miller BS; Petryk A
    Bone Marrow Transplant; 2008 Jun; 41(12):1005-11. PubMed ID: 18278070
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Incidence of Bloodstream Infections after Hematopoietic Stem Cell Transplantation for Hurler Syndrome.
    Dunseath C; O'Connor G; Mahulkar S; Badia P; Koo J; Dandoy CE
    Transplant Cell Ther; 2023 Nov; 29(11):707.e1-707.e4. PubMed ID: 37582469
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Haematopoietic stem cell gene therapy in inborn errors of metabolism.
    Chiesa R; Bernardo ME
    Br J Haematol; 2022 Jul; 198(2):227-243. PubMed ID: 35535965
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Open issues in Mucopolysaccharidosis type I-Hurler.
    Parini R; Deodato F; Di Rocco M; Lanino E; Locatelli F; Messina C; Rovelli A; Scarpa M
    Orphanet J Rare Dis; 2017 Jun; 12(1):112. PubMed ID: 28619065
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans.
    Scala S; Basso-Ricci L; Dionisio F; Pellin D; Giannelli S; Salerio FA; Leonardelli L; Cicalese MP; Ferrua F; Aiuti A; Biasco L
    Nat Med; 2018 Nov; 24(11):1683-1690. PubMed ID: 30275570
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Effect of recombinant human growth hormone on changes in height, bone mineral density, and body composition over 1-2 years in children with Hurler or Hunter syndrome.
    Polgreen LE; Thomas W; Orchard PJ; Whitley CB; Miller BS
    Mol Genet Metab; 2014 Feb; 111(2):101-6. PubMed ID: 24368158
    [TBL] [Abstract][Full Text] [Related]  

  • 20.
    ; ; . PubMed ID:
    [No Abstract]   [Full Text] [Related]  

    [Next]    [New Search]
    of 7.