Pages that link to "Q39566137"
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The following pages link to Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer (Q39566137):
Displaying 50 items.
- Adeno-associated virus-mediated cancer gene therapy: current status (Q27026177) (← links)
- Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan (Q27300989) (← links)
- Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates (Q27318381) (← links)
- CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome) (Q27333793) (← links)
- Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy (Q30541880) (← links)
- Myeloid/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease (Q30554347) (← links)
- Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy (Q33649318) (← links)
- Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse (Q33732656) (← links)
- Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10 (Q33870720) (← links)
- Gene therapy for lysosomal storage disorders: a good start (Q34502978) (← links)
- Therapies of mucopolysaccharidosis IVA (Morquio A syndrome). (Q34545883) (← links)
- Lysosomal storage disease: gene therapy on both sides of the blood-brain barrier (Q35034401) (← links)
- Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood (Q35053456) (← links)
- Gene therapy for neurologic manifestations of mucopolysaccharidoses (Q35362251) (← links)
- Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase (Q35600316) (← links)
- Current gene therapy using viral vectors for chronic pain (Q35658521) (← links)
- Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice (Q35894587) (← links)
- Clinical applications involving CNS gene transfer (Q35899850) (← links)
- In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy (Q35999976) (← links)
- A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB. (Q36521756) (← links)
- Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease (Q36543422) (← links)
- Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector (Q36951265) (← links)
- Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery (Q36982214) (← links)
- Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC. (Q37305882) (← links)
- Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease. (Q37698337) (← links)
- Current and potential therapeutic strategies for mucopolysaccharidoses (Q38194635) (← links)
- Recent advances in gene therapy for lysosomal storage disorders (Q38546492) (← links)
- Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration (Q38703339) (← links)
- Viral vectors for therapy of neurologic diseases (Q38746238) (← links)
- Nanotechnology applied to treatment of mucopolysaccharidoses. (Q38850942) (← links)
- Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB. (Q38851110) (← links)
- Adeno-Associated Virus-Based Gene Therapy for CNS Diseases. (Q38856794) (← links)
- Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting (Q38874937) (← links)
- In vivo adeno-associated viral vector-mediated genetic engineering of white and brown adipose tissue in adult mice (Q39348746) (← links)
- Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice (Q39600788) (← links)
- Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID. (Q40277701) (← links)
- Variables influencing fluorimetric N-sulfoglucosamine sulfohydrolase (SGSH) activity measurement in brain homogenates (Q41102164) (← links)
- Toward a gene therapy for neurological and somatic MPSIIIA (Q41927384) (← links)
- Differential prevalence of antibodies against adeno-associated virus in healthy children and patients with mucopolysaccharidosis III: perspective for AAV-mediated gene therapy (Q41935298) (← links)
- Timing of Gene Therapy Interventions: The Earlier, the Better (Q41939107) (← links)
- Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy (Q42176546) (← links)
- Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery (Q42180849) (← links)
- High and prolonged sulfamidase secretion by the liver of MPS-IIIA mice following hydrodynamic tail vein delivery of antibiotic-free pFAR4 plasmid vector (Q42198901) (← links)
- Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. (Q45325768) (← links)
- Treatment of brain disease in the mucopolysaccharidoses (Q45874710) (← links)
- A GLP-compliant toxicology and biodistribution study: systemic delivery of a rAAV9 vector for the treatment of mucopolysaccharidosis IIIB. (Q45881431) (← links)
- Gene therapy for Mucopolysaccharidoses. (Q47558790) (← links)
- Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy (Q50420936) (← links)
- Recent progress and considerations for AAV gene therapies targeting the central nervous system. (Q54206545) (← links)
- Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice (Q58765137) (← links)