| P50 | author | Coy Heldermon | Q72441560 |
| P2093 | author name string | M D Bloom | |
| | J A Gilkes | |
| P2860 | cites work | Cross-dressing the virion: the transcapsidation of adeno-associated virus serotypes functionally defines subgroups | Q24623923 |
| | Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding alpha-N-acetylglucosaminidase | Q24647066 |
| | Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications | Q28201581 |
| | Development of sensory, motor and behavioral deficits in the murine model of Sanfilippo syndrome type B | Q28512711 |
| | Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB | Q33931583 |
| | Clades of Adeno-associated viruses are widely disseminated in human tissues. | Q34193756 |
| | Capsid serotype and timing of injection determines AAV transduction in the neonatal mice brain | Q34795088 |
| | Adeno-associated viral serotypes produce differing titers and differentially transduce neurons within the rat basal and lateral amygdala | Q35095825 |
| | Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. | Q35111738 |
| | Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors | Q35712206 |
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| | Efficient gene therapy-based method for the delivery of therapeutics to primate cortex | Q37114888 |
| | Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. | Q37181003 |
| | Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges | Q37866086 |
| | Mucopolysaccharidosis III (Sanfilippo Syndrome)- disease presentation and experimental therapies. | Q38262731 |
| | Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity | Q39686097 |
| | Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain | Q40329324 |
| | Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors | Q40692830 |
| | Convection-enhanced delivery and systemic mannitol increase gene product distribution of AAV vectors 5, 8, and 9 and increase gene product in the adult mouse brain | Q41333437 |
| | Convection-enhanced delivery of AAV2 in white matter--a novel method for gene delivery to cerebral cortex | Q42273399 |
| | Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain | Q42280576 |
| | Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. | Q43106648 |
| | 4-Methylumbelliferyl alpha-N-acetylglucosaminidase activity for diagnosis of Sanfilippo B disease | Q44319034 |
| | Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system | Q45007196 |
| | Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain | Q45420843 |
| | Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. | Q45864505 |
| | Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain | Q45866495 |
| | Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice | Q47643319 |
| P304 | page(s) | 48-54 | |
| P577 | publication date | 2015-12-07 | |
| P1433 | published in | Molecular Genetics and Metabolism Reports | Q27725363 |
| P1476 | title | Preferred transduction with AAV8 and AAV9 via thalamic administration in the MPS IIIB model: A comparison of four rAAV serotypes | |
| P478 | volume | 6 | |