Elizabeth Ramsburg

Administration of a barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transport

Yulia Dzhashiashvili, Jodi L. McBride, Emily Fabyanic, Xin Huang et autres

Adeno-associated viral vectors have become a leading choice for gene therapy in the central nervous system due to their safety profile, efficient neuronal transduction, and capacity for sustained transgene expression. We previously reported that AAV2-derived capsids developed using the BRAVE (Barcoded Rational …

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AAV-delivered PPT1 provides long-term neurological benefits in CLN1 mice and achieves therapeutic levels in sheep brain

Md. Suhail Alam†, Apeksha Khatiwada, Samantha L. Eaton, Daniel M. Cohen et autres

CLN1 disease is a fatal neurodegenerative condition caused by deficiency in palmitoyl-protein thioesterase 1 (PPT1), for which no disease-modifying therapy exists. The disease affects the entire central nervous system (CNS), necessitating widespread delivery of therapeutics to the brain and spinal cord. Adeno-associated …

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Administration of barcoded AAV capsid library to the putamen of non-human primates identifies variants with efficient retrograde transport

Yulia Dzhashiashvili, Jodi L. McBride, Emily Fabyanic, Xin Huang et autres

ABSTRACT Adeno-associated viral vectors have become a leading choice for gene therapy in the central nervous system due to their safety profile, efficient neuronal transduction, and capacity for sustained transgene expression. We previously reported that AAV2-derived capsids developed using the BRAVE (Barcoded …

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AAV-mediated gene therapy for focal epilepsy by expressing neuropeptide Y and Y2 receptor in rodent and non-human primate hippocampus

Esbjörn Melin, Pernilla Fagergren, David Dobry, Stefano Cattaneo et autres

Epilepsy affects approximately 50 million people worldwide, and over 30% of patients are considered treatment resistant to currently available anti-seizure drugs. Neuropeptide Y (NPY) has been shown to inhibit excitatory synaptic transmission in hippocampal slices from human epilepsy patients via Y2 receptors …

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Portable transcranial therapeutic ultrasound enhances targeted gene delivery for Parkinson’s disease: from rodent models to non-human primates

Alec J. Batts, Robin Ji, Sua Bae, Fotios N. Tsitsos et autres

ABSTRACT Gene therapy for neurodegenerative diseases faces significant challenges due to the blood-brain barrier (BBB), which limits drug delivery to the central nervous system (CNS). While clinical trials for Parkinson’s disease (PD) have progressed, administration of vectors expressing enzymatic or neurotrophic factor …

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Pathogen class-specific transcriptional responses derived from PBMCs accurately discriminate between fungal, bacterial, and viral infections

Julie M. Steinbrink, Yiling Liu, Ricardo Henao, Ephraim L. Tsalik et autres

Immune responses during acute infection often contain canonical elements which are shared across the responses to an array of agents within a given pathogen class (i.e., respiratory viral infection). Identification of these shared, canonical elements across similar infections offers the potential for …

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305. PBMC-Derived Transcriptomic Signatures Accurately Discriminate Between Viral, Bacterial, and Fungal Infections and can be Translated to Real-World Human Infections

Julie M. Steinbrink, Yiling Liu, Ricardo Henao, Ephraim L. Tsalik et autres

Abstract Background Analysis of host gene expression patterns (‘signatures’) can provide diagnostic information to determine the etiology of acute infection. In vitro experiments can be used to supplement our knowledge about these signatures. Methods We performed in vitro human peripheral blood mononuclear …

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Recombinant MVA-prime elicits neutralizing antibody responses by inducing antigen-specific B cells in the germinal center

Leila Eslamizar, Constantinos Petrovas, David J. Leggat, Kathryn L. Furr et autres

Abstract The RV144 HIV-1 vaccine trial has been the only clinical trial to date that has shown any degree of efficacy and associated with the presence of vaccine-elicited HIV-1 envelope-specific binding antibody and CD4+ T-cell responses. This trial also showed that a …

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Comparison of multiple vaccine vectors in a single heterologous prime-boost trial

Hua Liao, Natalie J. Thornburg, Elizabeth Ramsburg, Christopher Sample et autres

The prevention of infectious disease via prophylactic immunization is a mainstay of global public health efforts. Vaccine design would be facilitated by a better understanding of the type and durability of immune responses generated by different vaccine vectors. We report here the …

A Host Transcriptional Signature for Presymptomatic Detection of Infection in Humans Exposed to Influenza H1N1 or H3N2

Lawrence Carin, Minhua Chen, Christopher W. Woods, Seth W. Glickman et autres

There is great potential for host-based gene expression analysis to impact the early diagnosis of infectious diseases. In particular, the influenza pandemic of 2009 highlighted the challenges and limitations of traditional pathogen-based testing for suspected upper respiratory viral infection. We inoculated human …

Cell-based Assay to Study Antibody-mediated Tau Clearance by Microglia

Donata De Marco, Renske L. R. E. Taggenbrock, Rosa Crespo, Wouter Koudstaal et autres

Alzheimer's disease (AD) is a progressive neurodegenerative condition in which aggregated tau and amyloid proteins accumulate in the brain causing neuronal dysfunction which eventually leads to cognitive decline. Hyperphosphorylated tau aggregates in the neuron are believed to cause most of the pathology …

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Cell-based Assay to Study Antibody-mediated Tau Clearance by Microglia

Donata De Marco, Renske L. R. E. Taggenbrock, Rosa Crespo, Wouter Koudstaal et autres

Alzheimer's disease (AD) is a progressive neurodegenerative condition in which aggregated tau and amyloid proteins accumulate in the brain causing neuronal dysfunction which eventually leads to cognitive decline. Hyperphosphorylated tau aggregates in the neuron are believed to cause most of the pathology …

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