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Hung, C.

Publications and source records attributed to Hung, C..

5 recordsLinked to original sources

Technical Note: Focused ultrasound-mediated blood-brain barrier opening for delivery of LNP-packaged modRNA therapy in a mouse model of Niemann-Pick Disease Type C

Efficient delivery of molecular therapies to the central nervous system (CNS) remains a major barrier to treating neurogenetic disorders such as Niemann-Pick type C (NPC) disease. Focused ultrasound-mediated blood-brain barrier opening (FUS-BBBO) has emerged as a non-invasive strategy to enhance delivery of systemically administered therapeutics. In this study, we evaluated whether FUS-BBBO could enable delivery of lipid nanoparticle (LNP)-packaged modified mRNA (modRNA) to the cerebellum in an NPC mouse model. A pilot study in wild-type mice demonstrated successful FUS-mediated BBB opening, delivery of LNP-packaged GFP mRNA, and subsequent protein expression in the cerebellum. We then performed a controlled study in NPC mice comparing delivery of LNP-GFP and LNP-NPC modRNA using intravenous administration with and without FUS-BBBO. BBB opening was confirmed by contrast-enhanced MRI in FUS-treated animals. Quantitative PCR revealed the presence of GFP mRNA in the cerebellum following FUS-BBBO, whereas NPC mRNA was minimal or undetectable across groups. However, no GFP or NPC1 protein expression was detected in the cerebellum by western blot in any experimental group. Consistent with this, no therapeutic effect on Purkinje cell survival was observed. These results demonstrate that while FUS-BBBO reliably induces BBB opening and can facilitate limited delivery of LNP-packaged mRNA to the brain, this did not translate into detectable protein expression or therapeutic benefit in the NPC model under the conditions tested. This discrepancy between successful delivery in wild-type mice and lack of efficacy in diseased animals points to potential important biological and/or formulation-dependent barriers that must be addressed to enable effective CNS delivery of LNP-based mRNA therapies.

bioengineering↗

A preoptic circuit triggers rewarming from torpor

Torpor is an adaptive hypometabolic state that enables homeotherm to survive periods of energetic challenge. This strategy ranges from short bouts of daily torpor to prolonged hibernation. During torpor, animals markedly suppress metabolic rate, body temperature, heart rate, and respiration, while retaining the ability to rewarm. Torpor therefore comprises two critical transitions -entry into a hypometabolic state and active rewarming-both essential for organismal viability. Although neural mechanisms controlling torpor entry have begun to emerge, the circuits that initiate active rewarming and restore euthermia remain poorly defined. Here we identify corticotropin-releasing hormone (Crh)-expressing neurons in the anterodorsal preoptic area (ADP) as a key population for rewarming from fasting-induced torpor in mice. These neurons become active around natural rewarming and are selectively required to limit the depth and duration of torpor. Unlike pathways mediating acute cold defence, stress hyperthermia, or LPS-induced fever, this circuit is dedicated to promoting timely recovery to euthermia. Closed-loop optogenetic activation of ADPCrh neurons during torpor entry rapidly initiates rewarming, and thermographic recordings show that brown adipose tissue (BAT) thermogenesis precedes locomotor arousal. ADPCrh neurons are predominantly GABAergic and project monosynaptically to the lateral preoptic area, whose terminal activation is sufficient to increase body temperature and locomotor activity. Finally, we find robust activation of ADPCrh neurons during rewarming in a hibernator, suggesting conserved logic for exiting deep torpor. Together, our results define a discrete preoptic circuit that drives recovery from torpor and provide a framework for understanding and potentially controlling timely rewarming from profound hypothermia.

neuroscience↗

Lysosomal stress induces amyloid-β aggregate release and reactive transformation in human astrocytes

Astrocytes are essential for brain homeostasis and are involved in amyloid-{beta} (A{beta}) clearance, but whether they can produce and release A{beta} aggregates remains unclear. Using human iPSC-derived astrocytes, we show that astrocytes cell autonomously generate small, diffusible A{beta} aggregates under baseline conditions. By combining ultrasensitive single-molecule imaging (DNA-PAINT) and immunoassays, we detect intracellular aggregates and their release into the media. Notably, lysosomal membrane damage induced by L-leucyl-L-leucine methyl ester (LLOMe) significantly increases A{beta} aggregate secretion without altering their size or morphology. Transcriptomic analysis and cytokine profiling reveal that lysosomal stress triggers a reactive astrocyte phenotype marked by upregulation of inflammatory genes and secreted cytokines. These findings suggest that astrocytes are not merely passive A{beta} scavengers but can actively contribute to extracellular A{beta} accumulation under lysosomal stress. Our study highlights astrocytes as active players in Alzheimers disease pathology.

neuroscience↗

A Novel Pan-Proteome Array for High-Throughput Profiling of the Humoral Response to Treponema pallidum subsp. pallidum: a Pre-Clinical Study

BackgroundGiven the resurgence of syphilis, research endeavors to improve current assays for serological diagnosis and management of this disease are a priority. A proteome-scale platform for high-throughput profiling of the humoral response to Treponema pallidum (T. pallidum) proteins during infection could identify antigens suitable to ameliorate the performance and capabilities of treponemal tests (TTs), which may require weeks to become positive following infection, cannot distinguish between active and previously treated infections, or assess treatment response. Additionally, because infection-induced immunity is partially protective, profiling the response to T. pallidum outer membrane proteins (OMPs) could help select vaccine candidates. MethodsWe developed a pan-proteome array (PPA) based on the Nichols and SS14 strain complete proteomes and used it to define the IgM and IgG humoral response to 1,009 T. pallidum proteins in sera collected longitudinally from long-term infected rabbits, and from rabbits that were infected, treated, and re-infected. FindingsApproximately a third of the pathogens proteome was recognized in infected animals, with a marked IgG response detectable between day-10 and day-20 post-infection. We found early, gradual, and late IgG kinetic profiles, strain-dependent differences in humoral reactivity, and post-treatment fluctuation in reactivity for several antigens. Very few antigens elicited an IgM response. Several OMPs were significantly and differentially recognized, but few elicited a robust response. InterpretationThe PPA allowed the identification of antigens that could facilitate early diagnosis and of a core set of OMP that could explain protection upon re-infection. No antigen appeared suitable to monitor treatment response. FundingNIH SBIR-R43AI149804 RESEARCH IN CONTEXTO_ST_ABSEvidence before this studyC_ST_ABSIn April 2024, we searched the PubMed database for articles on preclinical studies using high throughput proteome arrays containing at least 10% of the predicted T. pallidum proteome that aimed at identifying antibody reactivity to T. pallidum antigens during experimental syphilis infection. We could retrieve only one manuscript. In this work, an array containing the T. pallidum partial proteome as annotated in the first sequenced Nichols strain genome (GCA_000008605.1) in 1998 was assembled using recombinant antigens expressed in Escherichia coli (E. coli). The resulting array was probed using pooled sera from three rabbits infected with the Nichols stain of T. pallidum, attained from infected animals at five time points following intratesticular infection. The small number of reactive antigens (n = 106) identified in this early study was likely to be an incomplete set of all antigens recognized during infection because not all the predicted targets in the T. pallidum proteome were successfully expressed and tested. In retrospect, additional limitations of the study included an initial suboptimal annotation of the Nichols genome used to define the pathogens proteome, which has now changed with the availability of a re-sequenced Nichols strain genome devoid of sequencing errors that affected the initial annotation process, and the refinement of bioinformatic pipelines for the identification of open reading frames (ORFs). Furthermore (as acknowledged by the authors), the possible presence of amplification errors in their expression clones might have affected the sequence of some protein targets and antibody binding to the targets. As a result, some of the T. pallidum antigens known to elicit a robust humoral response during experimental infection were not detected in this antigenic screen. Lastly, employing only the Nichols strain in this early study did not consider that a significant portion of the circulating syphilis strains belong to the SS14 clade of T. pallidum. Added value of this studyThis novel PPA, combined with a more robust experiential design than ever reported, allowed us to overcome most of the limitations associated with the study mentioned above, as we were able to a) use the most recent annotations for the selected T. pallidum strains based on accurate genome sequences, b) print the pathogens virtually complete proteome in the study array, c) analyze individual sera to account for rabbit-to-rabbit variability in the humoral response to infection rather than pooled sera, d) detect both IgM and IgG over 10 or 20 timepoints, depending on the experimental design, e) obtain information on how the humoral response evolved upon treatment and re-infection and, finally, f) evaluate all of the above in animals infected with two T. pallidum strains whose genetic background is representative of the two currently circulating clades of the syphilis agent. Implications of all the available evidenceOur study provides new and more comprehensive data on how humoral immunity for two classes of antibodies develops during infection and how it evolves in response to treatment and re-infection. The analysis of sera collected at tightly spaced time points post-inoculation and for an extensive period post-infection provides a wealth of information to improve the diagnostic performance of existing tests detecting treponemal antigens. The analysis of differential immunity specific to the pathogens putative OMPs provides a rationale for vaccine candidate selection.

microbiology↗

Age-Associated Weaker Immunity to Coronaviruses is Characteristic of Children that Develop Multisystem Inflammatory Syndrome following SARS-CoV-2 Infection

We analyzed the antibody and cytokine responses of twenty-three patients with multisystem inflammatory syndrome of children (MIS-C) that appeared with a three-to-six-week delay following a mild or asymptomatic SARS-CoV-2 infection. These responses were compared to healthy convalescent pediatric COVID-19 patients approximately twenty-eight days after the onset of symptoms. Both groups had strong IgG responses to SARS-CoV-2 spike (S) and nucleocapsid (N) proteins, but the MIS-C patients had weaker antibody responses to certain epitopes in the SARS-CoV-2 S and N proteins and to the S and N proteins of endemic human coronaviruses (HCoV) compared to pediatric convalescent COVID patients. HCoV antibody reactivity was correlated with age. In contrast, MIS-C patients had elevated serum levels of several proinflammatory cytokines compared to convalescent COVID patients, including interleukins IL-6, IL-8, IL-18 and chemokines CCL2, CCL8, CXCL5, CXCL9 and CXCL10 as well as tumor necrosis factor alpha and interferon gamma. Moreover, many cytokine responses of MIS-C patients were positively correlated with antibody responses to the SARS-CoV-2 S, N, membrane and ORF3a proteins while pediatric convalescent COVID patient cytokine responses were more often negatively correlated with antibody responses to the S, N and ORF3a proteins of SARS-CoV-2.

immunology↗