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2. Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I. Gomez-Ospina N; Scharenberg SG; Mostrel N; Bak RO; Mantri S; Quadros RM; Gurumurthy CB; Lee C; Bao G; Suarez CJ; Khan S; Sawamoto K; Tomatsu S; Raj N; Attardi LD; Aurelian L; Porteus MH Nat Commun; 2019 Sep; 10(1):4045. PubMed ID: 31492863 [TBL] [Abstract][Full Text] [Related]
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4. [Molecular diagnosis and gene therapy for Gaucher disease]. Ohashi T Nihon Rinsho; 1993 Sep; 51(9):2300-7. PubMed ID: 8411706 [TBL] [Abstract][Full Text] [Related]
5. High levels of human glucocerebrosidase activity in macrophages of long-term reconstituted mice after retroviral infection of hematopoietic stem cells. Correll PH; Colilla S; Dave HP; Karlsson S Blood; 1992 Jul; 80(2):331-6. PubMed ID: 1627794 [TBL] [Abstract][Full Text] [Related]
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8. Detection and isolation of gene-corrected cells in Gaucher disease via a fluorescence-activated cell sorter assay for lysosomal glucocerebrosidase activity. Lorincz M; Herzenberg LA; Diwu Z; Barranger JA; Kerr WG Blood; 1997 May; 89(9):3412-20. PubMed ID: 9129049 [TBL] [Abstract][Full Text] [Related]
9. The presence of an autologous marrow stromal cell layer increases glucocerebrosidase gene transduction of long-term culture initiating cells (LTCICs) from the bone marrow of a patient with Gaucher disease. Wells S; Malik P; Pensiero M; Kohn DB; Nolta JA Gene Ther; 1995 Oct; 2(8):512-20. PubMed ID: 8593601 [TBL] [Abstract][Full Text] [Related]
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11. Correction of glucocerebrosidase deficiency after retroviral-mediated gene transfer into hematopoietic progenitor cells from patients with Gaucher disease. Fink JK; Correll PH; Perry LK; Brady RO; Karlsson S Proc Natl Acad Sci U S A; 1990 Mar; 87(6):2334-8. PubMed ID: 2315324 [TBL] [Abstract][Full Text] [Related]
12. Correction of the enzyme deficiency in hematopoietic cells of Gaucher patients using a clinically acceptable retroviral supernatant transduction protocol. Xu L; Stahl SK; Dave HP; Schiffmann R; Correll PH; Kessler S; Karlsson S Exp Hematol; 1994 Feb; 22(2):223-30. PubMed ID: 8299741 [TBL] [Abstract][Full Text] [Related]
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