| P2093 | author name string | Andrew Bird | |
| | Dwight D Koeberl | |
| | Baodong Sun | |
| | Haoyue Zhang | |
| | Sarah P Young | |
| | Songtao Li | |
| P2860 | cites work | Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease. | Q50736816 |
| | The spectrum and diagnosis of acid maltase deficiency. | Q54261710 |
| | Abnormal trafficking of sarcolemmal proteins in alpha-glucosidase deficiency | Q73096464 |
| | Characterization of the human lysosomal alpha-glucosidase gene | Q24529537 |
| | Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease | Q28277595 |
| | Sequence of the cDNA and 5'-flanking region for human acid alpha-glucosidase, detection of an intron in the 5' untranslated leader sequence, definition of 18-bp polymorphisms, and differences with previous cDNA and amino acid sequences | Q28299489 |
| | Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme | Q28511802 |
| | Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy | Q28586585 |
| | Modulation of disease severity in mice with targeted disruption of the acid alpha-glucosidase gene | Q28587632 |
| | Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II | Q28592264 |
| | Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial | Q33941289 |
| | Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A. | Q34595095 |
| | Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase | Q35586463 |
| | Murine acid alpha-glucosidase: cell-specific mRNA differential expression during development and maturation | Q35786827 |
| | Enzyme replacement and enhancement therapies for lysosomal diseases | Q35800534 |
| | Correction of multiple striated muscles in murine Pompe disease through adeno-associated virus-mediated gene therapy | Q37161996 |
| | Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease | Q37219555 |
| | Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II. | Q37259816 |
| | Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up | Q39218884 |
| | Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. | Q40483404 |
| | Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers. | Q40483409 |
| | Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver | Q41753510 |
| | Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice | Q43628691 |
| | A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease | Q44312356 |
| | Analysis of a glucose tetrasaccharide elevated in Pompe disease by stable isotope dilution–electrospray ionization tandem mass spectrometry | Q44414194 |
| | Enzyme replacement therapy in the mouse model of Pompe disease | Q44624370 |
| | Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice | Q45071217 |
| | Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. | Q45259698 |
| | Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter | Q45457148 |
| | Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II. | Q45513180 |
| | Packaging of an AAV vector encoding human acid α-glucosidase for gene therapy in glycogen storage disease type II with a modified hybrid adenovirus-AAV vector | Q45862016 |
| | Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice | Q45877667 |
| | Glycogen storage in multiple muscles of old GSD-II mice can be rapidly cleared after a single intravenous injection with a modified adenoviral vector expressing hGAA. | Q45879179 |
| | Glucose tetrasaccharide as a biomarker for monitoring the therapeutic response to enzyme replacement therapy for Pompe disease | Q46484079 |
| P433 | issue | 10 | |
| P921 | main subject | vector-borne disease | Q2083837 |
| P304 | page(s) | 913-920 | |
| P577 | publication date | 2009-10-01 | |
| P1433 | published in | Journal of Gene Medicine | Q15746377 |
| P1476 | title | Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression | |
| P478 | volume | 11 | |