| scholarly article | Q13442814 |
| P6179 | Dimensions Publication ID | 1052936104 |
| P356 | DOI | 10.1023/A:1005303331218 |
| P2093 | author name string | Y. Tan | |
| C.C. Chang | |||
| C.M. Lin | |||
| K.J. Hsiao | |||
| Y.M. Lee | |||
| P2860 | cites work | Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer | Q28115950 |
| A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia | Q28291035 | ||
| High-efficiency retroviral-mediated gene transfer into human and nonhuman primate peripheral blood lymphocytes | Q34003623 | ||
| Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells | Q34073981 | ||
| Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction | Q34354588 | ||
| Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency | Q34454454 | ||
| Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver | Q35559973 | ||
| Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses | Q35837026 | ||
| THE STRUCTURE OF THE PHENYLALANINE-HYDROXYLATION COFACTOR | Q36402585 | ||
| Peripheral blood progenitors as a target for genetic correction of p47phox-deficient chronic granulomatous disease | Q36470854 | ||
| Expression of human alpha 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes | Q36757603 | ||
| Efficient transfer and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector | Q37319132 | ||
| Human dihydropteridine reductase: a method for the measurement of activity in cultured cells, and its application to malignant hyperphenylalaninemia | Q39286742 | ||
| Inborn errors of pterin metabolism | Q39548627 | ||
| Maternal phenylketonuria and hyperphenylalaninemia. An international survey of the outcome of untreated and treated pregnancies | Q40364201 | ||
| Second-generation adenovirus vectors | Q40939190 | ||
| Somatic gene therapy for phenylketonuria and other hepatic deficiencies | Q41180797 | ||
| Molecular analysis of T lymphocyte-directed gene therapy for adenosine deaminase deficiency: long-term expression in vivo of genes introduced with a retroviral vector | Q41192415 | ||
| Assessment of recombinant adenoviral vectors for hepatic gene therapy | Q43425277 | ||
| Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice | Q43836200 | ||
| Improved gene transfer into human lymphocytes using retroviruses with the gibbon ape leukemia virus envelope | Q45767602 | ||
| Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients | Q45866834 | ||
| T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. | Q45866838 | ||
| Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. | Q45867442 | ||
| Preclinical studies of lymphocyte gene therapy for mild Hunter syndrome (mucopolysaccharidosis type II). | Q45878645 | ||
| Reconstitution of enzymatic activity in hepatocytes of phenylalanine hydroxylase-deficient mice | Q45878913 | ||
| Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo | Q45879739 | ||
| Long-Term Improvement of Hypercholesterolemia After Ex Vivo Gene Therapy in LDLR-Deficient Rabbits | Q45880751 | ||
| Effect of stopping low-phenylalanine diet on intellectual progress of children with phenylketonuria | Q49313601 | ||
| Treatment of phenylketonuria with a diet low in phenylalanine. | Q52172881 | ||
| Genetics and biochemistry of the phenylketonuria-present state | Q66972930 | ||
| Prenatal diagnosis of "dihydrobiopterin synthetase" deficiency, a variant form of phenylketonuria | Q68908457 | ||
| Intelligence and personality characteristics in adults with untreated atypical phenylketonuria and mild hyperphenylalaninemia | Q70678284 | ||
| Umbilical cord blood transplantation | Q71948181 | ||
| P433 | issue | 6 | |
| P304 | page(s) | 742-754 | |
| P577 | publication date | 1997-01-01 | |
| P1433 | published in | Journal of Inherited Metabolic Disease | Q6295359 |
| P478 | volume | 20 |
| Q46536688 | Characterization of transgenic mice with the expression of phenylalanine hydroxylase and GTP cyclohydrolase I in the skin |
| Q44123143 | Comparison of epidermal keratinocytes and dermal fibroblasts as potential target cells for somatic gene therapy of phenylketonuria |
| Q40827772 | Development of a skin-based metabolic sink for phenylalanine by overexpression of phenylalanine hydroxylase and GTP cyclohydrolase in primary human keratinocytes |
| Q37221695 | Expression of phenylalanine hydroxylase (PAH) in erythrogenic bone marrow does not correct hyperphenylalaninemia in Pahenu2 mice |
| Q34325086 | Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer |
| Q45423981 | Reversal of gene expression profile in the phenylketonuria mouse model after adeno-associated virus vector-mediated gene therapy |
| Q42809966 | Towards metabolic sink therapy for mut methylmalonic acidaemia: correction of methylmalonyl-CoA mutase deficiency in T lymphocytes from a mut methylmalonic acidaemia child by retroviral-mediated gene transfer |
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