| scholarly article | Q13442814 |
| review article | Q7318358 |
| P356 | DOI | 10.1159/000072456 |
| P953 | full work available at URL | https://www.karger.com/Article/Pdf/72456 |
| P698 | PubMed publication ID | 14583667 |
| P2093 | author name string | Alessandra D'Azzo | |
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| Retrovirus vector-mediated correction and cross-correction of lysosomal alpha-mannosidase deficiency in human and feline fibroblasts | Q40948688 | ||
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| Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of β-glucuronidase | Q41102578 | ||
| Expression of human beta-hexosaminidase alpha-subunit gene (the gene defect of Tay-Sachs disease) in mouse brains upon engraftment of transduced progenitor cells | Q41212883 | ||
| Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain | Q41358776 | ||
| Biology and potential strategies for the treatment of GM2 gangliosidoses | Q41757590 | ||
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| The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors. | Q42827486 | ||
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| Four year follow-up of a case of fucosidosis treated with unrelated donor bone marrow transplantation | Q43613031 | ||
| Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction | Q43699928 | ||
| Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements | Q43722399 | ||
| Gene transfer to the nigrostriatal system by hybrid herpes simplex virus/adeno-associated virus amplicon vectors | Q43822089 | ||
| Long-term delivery of a lysosomal enzyme by genetically modified fibroblasts in dogs | Q44065456 | ||
| Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. | Q44267005 | ||
| Transduction of fibroblasts and CD34+ progenitors using a selectable retroviral vector containing cDNAs encoding arylsulfatase A and CD24. | Q44864430 | ||
| Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses | Q45749766 | ||
| Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice. | Q45751235 | ||
| Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse | Q45856025 | ||
| Viral receptors and vector purification: new approaches for generating clinical-grade reagents | Q45857978 | ||
| Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation | Q45858480 | ||
| Gene therapy for canine alpha-L-iduronidase deficiency: in utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease | Q45859200 | ||
| Combined ultrafiltration-transduction in a hollow-fiber bioreactor facilitates retrovirus-mediated gene transfer into peripheral blood lymphocytes from patients with mucopolysaccharidosis type II. | Q45863806 | ||
| Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector | Q45869639 | ||
| Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann-Pick disease | Q45872484 | ||
| Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII. | Q45873416 | ||
| Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors | Q45873434 | ||
| In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice | Q45875261 | ||
| Stable transduction with lentiviral vectors and amplification of immature hematopoietic progenitors from cord blood of preterm human fetuses | Q45875425 | ||
| Canine fucosidosis: a model for retroviral gene transfer into haematopoietic stem cells | Q45883167 | ||
| Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B. | Q45981487 | ||
| Enzyme-replacement therapy in mucopolysaccharidosis I. | Q48705361 | ||
| Nonablative neonatal marrow transplantation attenuates functional and physical defects of beta-glucuronidase deficiency. | Q48975078 | ||
| Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. | Q53912470 | ||
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| In utero transplantation of fetal liver cells in the mucopolysaccharidosis type VII mouse results in low-level chimerism, but overexpression of beta-glucuronidase can delay onset of clinical signs | Q73586038 | ||
| Human gene marker/therapy clinical protocols | Q73707811 | ||
| Hematopoietic stem-cell transplantation in globoid-cell leukodystrophy | Q74450560 | ||
| Biochemical, pathological, and clinical response to transplantation of normal bone marrow cells into acid sphingomyelinase-deficient mice | Q74493983 | ||
| P433 | issue | 2-3 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | gene delivery | Q2790163 |
| P304 | page(s) | 71-85 | |
| P577 | publication date | 2003-01-01 | |
| P1433 | published in | Acta Haematologica | Q15749687 |
| P1476 | title | Gene transfer strategies for correction of lysosomal storage disorders | |
| Gene Transfer Strategies for Correction of Lysosomal Storage Disorders | |||
| P478 | volume | 110 |
| Q81058015 | AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease |
| Q91584322 | Bone marrow transplantation for lysosomal storage disorders |
| Q42194288 | Chemokine-induced recruitment of genetically modified bone marrow cells into the CNS of GM1-gangliosidosis mice corrects neuronal pathology |
| Q45628046 | Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene |
| Q37686232 | Deregulated sphingolipid metabolism and membrane organization in neurodegenerative disorders. |
| Q36362402 | Gangliosides as apoptotic signals in ER stress response |
| Q34556549 | Gaucher disease: pathological mechanisms and modern management |
| Q36592822 | Genetic diseases of sphingolipid metabolism: pathological mechanisms and therapeutic options |
| Q34741419 | In utero therapy for congenital disorders using amniotic fluid stem cells |
| Q37826106 | Secondary alterations of sphingolipid metabolism in lysosomal storage diseases. |
| Q42616221 | Sphingolipid signalling and liver diseases |
| Q35825859 | The cell biology of lysosomal storage disorders |
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