ArticleHuman gene therapy2010
Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders.
Article in Human gene therapy, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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Who cites it
27 citing papers in PubMed, 42 citations in OpenAlex.
- Review
- A Preliminary Study on the Accuracy of MRI-Guided Thalamic Infusion of AAV2-GFP and Biodistribution Analysis Using Cryo-Fluorescence Tomography in Nonhuman Primates.Pharmaceutics · 2025Article
- Pre-clinical delivery of gene therapy products to the cerebrospinal fluid: challenges and considerations for clinical translation.Frontiers in molecular neuroscience · 2023Review
- Current advances in gene therapy of mitochondrial diseases.Journal of translational medicine · 2022Review
- Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates.Human gene therapy · 2020Article
- Development of a novel frameless skull-mounted ball-joint guide array for use in image-guided neurosurgery.Journal of neurosurgery · 2020Article
- Adeno-associated viral vector serotype 9-based gene therapy for Niemann-Pick disease type A.Science translational medicine · 2019Article
- Kinetics and MR-Based Monitoring of AAV9 Vector Delivery into Cerebrospinal Fluid of Nonhuman Primates.Molecular therapy. Methods & clinical development · 2019Article
- MR-guided delivery of AAV2-BDNF into the entorhinal cortex of non-human primates.Gene therapy · 2018Article
- Imaging of Convective Drug Delivery in the Nervous System.Neurosurgery clinics of North America · 2017Review
- A Unilateral Cervical Spinal Cord Contusion Injury Model in Non-Human Primates (Macaca mulatta).Journal of neurotrauma · 2016Article
- Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.Methods in molecular biology (Clifton, N.J.) · 2016Review
- Gene therapy for the neurological manifestations in lysosomal storage disorders.Journal of lipid research · 2014Review
- Article
- Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Safety study of adeno-associated virus serotype 2-mediated human acid sphingomyelinase expression in the nonhuman primate brain.Human gene therapy · 2012Article
- Gene-based therapy of Parkinson's Disease: Translation from animal model to human clinical trial employing convection enhanced delivery.Annals of neurosciences · 2012Review
- Guided delivery of adeno-associated viral vectors into the primate brain.Advanced drug delivery reviews · 2012Review
- Lentiviral-mediated gene transfer to the sheep brain: implications for gene therapy in Batten disease.Human gene therapy · 2011Article
Corrections and comments
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Authors and funding
12 authors at 1 institution in 1 country.
Funding
Abstract
Gene replacement therapy for the neurological deficits caused by lysosomal storage disorders, such as in Niemann-Pick disease type A, will require widespread expression of efficacious levels of acid sphingomyelinase (ASM) in the infant human brain. At present there is no treatment available for this devastating pediatric condition. This is partly because of inherent constraints associated with the efficient delivery of therapeutic agents into the CNS of higher order models. In this study we used an adeno-associated virus type 2 (AAV2) vector encoding human acid sphingomyelinase tagged with a viral hemagglutinin epitope (AAV2-hASM-HA) to transduce highly interconnected CNS regions such as the brainstem and thalamus. On the basis of our data showing global cortical expression of a secreted reporter after thalamic delivery in nonhuman primates (NHPs), we set out to investigate whether such widespread expression could be enhanced after brainstem infusion. To maximize delivery of the therapeutic transgene throughout the CNS, we combined a single brainstem infusion with bilateral thalamic infusions in naive NHPs. We found that enzymatic augmentation in brainstem, thalamic, cortical, as well subcortical areas provided convincing evidence that much of the large NHP brain can be transduced with as few as three injection sites.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.