↓ Direkt till sidans innehåll
↓ Direkt till sidans sekundära innehåll (sidomenyn)

Träfflista för sökning "WFRF:(Gaspar H. Bobby) "

Sökning: WFRF:(Gaspar H. Bobby)

Resultat 1-5 av 5

Sortera/gruppera träfflistan

Sortering: Träffar per sida:

Numrering	Referens	Omslagsbild	Hitta
1.	Güngör, Tayfun, et al. (författare) Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. 2014 Ingår i: Lancet. - 1474-547X. ; 383:9915, s. 436-448 Tidskriftsartikel (refereegranskat)abstract In chronic granulomatous disease allogeneic haemopoietic stem-cell transplantation (HSCT) in adolescents and young adults and patients with high-risk disease is complicated by graft-failure, graft-versus-host disease (GVHD), and transplant-related mortality. We examined the effect of a reduced-intensity conditioning regimen designed to enhance myeloid engraftment and reduce organ toxicity in these patients.
2.	Debnath, Shubhranshu, et al. (författare) Lentiviral Vectors with Cellular Promoters Correct Anemia and Lethal Bone Marrow Failure in a Mouse Model for Diamond-Blackfan Anemia 2017 Ingår i: Molecular Therapy. - : Elsevier BV. - 1525-0016. ; 25:8, s. 1805-1814 Tidskriftsartikel (refereegranskat)abstract Diamond-Blackfan anemia is a congenital erythroid hypoplasia and is associated with physical malformations and a predisposition to cancer. Twenty-five percent of patients with Diamond-Blackfan anemia have mutations in a gene encoding ribosomal protein S19 (RPS19). Through overexpression of RPS19 using a lentiviral vector with the spleen focus-forming virus promoter, we demonstrated that the Diamond-Blackfan anemia phenotype can be successfully treated in Rps19-deficient mice. In our present study, we assessed the efficacy of a clinically relevant promoter, the human elongation factor 1α short promoter, with or without the locus control region of the β-globin gene for treatment of RPS19-deficient Diamond-Blackfan anemia. The findings demonstrate that these vectors rescue the proliferation defect and improve erythroid development of transduced RPS19-deficient bone marrow cells. Remarkably, bone marrow failure and severe anemia in Rps19-deficient mice was cured with enforced expression of RPS19 driven by the elongation factor 1α short promoter. We also demonstrate that RPS19-deficient bone marrow cells can be transduced and these cells have the capacity to repopulate bone marrow in long-term reconstituted mice. Our results collectively demonstrate the feasibility to cure RPS19-deficient Diamond-Blackfan anemia using lentiviral vectors with cellular promoters that possess a reduced risk of insertional mutagenesis. Diamond-Blackfan anemia is a congenital erythroid hypoplasia. Twenty-five percent of patients have mutations in a gene encoding ribosomal protein S19. Using an RPS19-deficient mouse model, Debnath et al. demonstrate the feasibility to cure RPS19-deficient Diamond-Blackfan anemia by means of lentiviral vectors with cellular promoters that possess a reduced risk of insertional mutagenesis.
3.	Gennery, Andrew R, et al. (författare) Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? 2010 Ingår i: The Journal of allergy and clinical immunology. - : Elsevier BV. - 1097-6825 .- 0091-6749. ; 126:3 Tidskriftsartikel (refereegranskat)abstract Hematopoietic stem cell transplantation remains the only treatment for most patients with severe combined immunodeficiencies (SCIDs) or other primary immunodeficiencies (non-SCID PIDs).
4.	Pan-Hammarström, Qiang, et al. (författare) Reexamining the role of TACI coding variants in common variable immunodeficiency and selective IgA deficiency 2007 Ingår i: NATURE GENETICS. - : Springer Science and Business Media LLC. - 1061-4036 .- 1546-1718. ; 39:4, s. 429-430 Tidskriftsartikel (refereegranskat)
5.	Pan-Hammarström, Qiang, et al. (författare) Reexamining the role of TACI coding variants in common variable immunodeficiency and selective IgA deficiency. 2007 Ingår i: Nature Genetics. - 1061-4036 .- 1546-1718. ; 39:4, s. 429-430 Tidskriftsartikel (övrigt vetenskapligt/konstnärligt)

Skapa referenser, mejla, bekava och länka

Länka till träfflistan

Resultat 1-5 av 5

Avgränsa träffmängd

Typ av publikation: tidskriftsartikel (5)

Typ av innehåll: refereegranskat (4); övrigt vetenskapligt/konstnärligt (1)

Författare/redaktör: Gaspar, H. Bobby (5); Du, Likun (2); Hammarström, Lennart (2); Pan-Hammarström, Qia ... (2); Fasth, Anders, 1945 (2); Fischer, Alain (2); visa fler...; Behrens, Timothy W (2); Cunningham-Rundles, ... (2); Güngör, Tayfun (2); Veys, Paul (2); Grimbacher, Bodo (2); Seger, Reinhard (2); Ahmad, Imran (1); Flygare, Johan (1); Albert, Michael H (1); Hoenig, Manfred (1); Belohradsky, Bernd H (1); Karlsson, Stefan (1); Hassan, Moustapha (1); Stepensky, Polina (1); Notarangelo, Luigi D (1); Neven, Bénédicte (1); Chen, Jun (1); Dahl, Maria (1); Björkander, Janne, 1 ... (1); Rentsch, Katharina (1); Blanche, Stéphane (1); Siva, Kavitha (1); Stussi, Georg (1); Gruhn, Bernd (1); Sedlacek, Petr (1); Schlegel, Paul G (1); Debnath, Shubhranshu (1); Rothe, Michael (1); Schambach, Axel (1); Jaako, Pekka (1); Gennery, Andrew R (1); Gennery, Andrew (1); Wulffraat, Nico M. (1); Slatter, Mary A (1); Grandin, Laure (1); Taupin, Pierre (1); Cant, Andrew J (1); Amrolia, Persis J (1); Davies, E Graham (1); Friedrich, Wilhelm (1); Mazzolari, Evelina (1); Porta, Fulvio (1); Bredius, Robbert G M (1); Lankester, Arjen C (1); visa färre...

Lärosäte: Göteborgs universitet (3); Linköpings universitet (1); Lunds universitet (1); Karolinska Institutet (1)

Språk: Engelska (5)

Forskningsämne (UKÄ/SCB): Medicin och hälsovetenskap (3); Naturvetenskap (1)

År

Kungliga biblioteket hanterar dina personuppgifter i enlighet med EU:s dataskyddsförordning (2018), GDPR. Läs mer om hur det funkar här.
Så här hanterar KB dina uppgifter vid användning av denna tjänst.

Copyright © LIBRIS - Nationella bibliotekssystem
LIBRIS.kb.se

pil uppåt

Stäng

Kopiera och spara länken för att återkomma till aktuell vy