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Biology subjects

Quach, B.

Publications and source records attributed to Quach, B..

3 recordsLinked to original sources

Dynamic, single-cell monitoring of CAR T cell identity and activation with Raman spectroscopy

Chimeric antigen receptor (CAR) T cell therapies have reshaped treatment for cancers and immune-mediated diseases, yet their safety and efficacy depend on both the proliferation of engineered cells and their dynamic functional state -- features that remain challenging to monitor in real-time clinical settings. Current methods require targeted labels, extensive processing, and provide only static snapshots of cell identity and activation. Here, we introduce a surface-enhanced Raman spectroscopy (SERS) and machine learning (ML) approach that enables single-cell identification of engineered CAR T cells without molecularly-targeted labels and time-resolved, semi-continuous monitoring of their functional activation state through a single physical readout spanning donor-derived cells and patient blood. From intrinsic vibrational signatures of live cells, we detect spectral differences resulting from engineered receptor expression in donor-derived CD19- and GD2-targeted CAR T cells (nine and five donors, respectively) with 81-85% donor-level accuracy, and resolve dynamic antigen-specific activation trajectories with temporal precision. Applying this same SERS-ML method to longitudinal samples from a four-patient CD19-CAR T therapy cohort, we classify patient peripheral blood mononuclear cells across pre-and post-infusion timepoints with a mean patient-level accuracy of 84%, and show that isolated CAR-positive T cells are distinguishable from both CAR-negative T cells and background populations with average 86-88% accuracies. These capabilities stem from biochemical signatures consistent with processes such as receptor expression, tonic signalling, and immune synapse formation, demonstrating a single method that reports both cellular identity and activation state with biochemical specificity across cell engineering and clinical monitoring contexts. Our results extend CAR T cell monitoring beyond static phenotyping and support the potential of SERS-ML analysis for rapid, point-of-care assessment of engineered immune cells across the therapeutic lifecycle.

bioengineering↗

Anti-Sense Oligonucleotide as a Therapeutic for Synucleinopathies: Pharmacokinetic, Safety and Efficacy Evaluation

Effective blood-brain barrier (BBB) penetration is a significant challenge for antisense oligonucleotide (ASO) therapies targeting neurodegenerative diseases. We utilized a peptide (ApoB11) mediated transport delivery of an ASO to the CNS following systemic delivery to reduce expression of targeted transcripts for neurodegenerative diseases. This study evaluates the pharmacokinetics, CNS penetration, and therapeutic efficacy of ApoB11:2-OMe ASO--Syn, an ASO for -synuclein (-Syn) suppression in synucleinopathies. After a single intraperitoneal (IP) injection (2 mg/kg) in C57BL/6 mice, ApoB11:ASO--Syn showed robust brain penetration, reaching peak concentrations (Cmax = 0.14 nMol/mg) at 1.5 hours and an extended brain half-life (t1/2 = 646.2 hours), indicating prolonged CNS retention. Immunofluorescence confirmed widespread uptake in neurons and endothelial cells. The ASO also accumulated in the liver (Cmax = 419.5 nMol/mg, t1/2 = 104.9 hours), consistent with receptor-mediated uptake. Acute and subacute toxicity studies revealed no systemic toxicity at the highest non-lethal dose (32 mg/kg). In a mouse model of dementia with lewy body (DLB) mice overexpressing human -Syn, ApoB11:ASO--Syn reduced -Syn mRNA and protein levels in the hippocampus and cortex by [~]50% at 16 mg/kg. These results demonstrate that ApoB11 is an effective ASO carrier for CNS delivery, supporting its potential as a therapeutic strategy for synucleinopathies. Graphical Abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=154 SRC="FIGDIR/small/651722v1_ufig1.gif" ALT="Figure 1"> View larger version (26K): org.highwire.dtl.DTLVardef@f73353org.highwire.dtl.DTLVardef@14ababforg.highwire.dtl.DTLVardef@12a92e3org.highwire.dtl.DTLVardef@1176b4_HPS_FORMAT_FIGEXP M_FIG C_FIG

pharmacology and toxicology↗

Epigenome-wide analysis uncovers a blood-based DNA methylation biomarker of lifetime cannabis use.

Cannabis use is highly prevalent and is associated with adverse and beneficial effects. To better understand the full spectrum of health consequences, biomarkers that accurately classify cannabis use are needed. DNA methylation (DNAm) is an excellent candidate, yet no blood-based epigenome-wide association studies (EWAS) in humans exist. We conducted an EWAS of lifetime cannabis use (ever vs. never) using blood-based DNAm data from a case-cohort study within Sister Study, a prospective cohort of women at risk of developing breast cancer (Discovery N=1,730 [855 ever users]; Replication N=853 [392 ever users]). We identified and replicated an association with lifetime cannabis use at cg15973234 (CEMIP): combined P=3.3x10-8. We found no overlap between published blood-based cis-meQTLs of cg15973234 and reported lifetime cannabis use-associated SNPs (P<0.05), suggesting that the observed DNAm difference was driven by cannabis exposure. We also developed a multi-CpG classifier of lifetime cannabis use using penalized regression of top EWAS CpGs. The resulting 49-CpG classifier produced an area under the curve (AUC)=0.74 (95% confidence interval [0.72, 0.76], P=2.00x10-5) in the discovery sample and AUC=0.54 ([0.51, 0.57], P=4.64x10-2) in the replication sample. Our EWAS findings provide evidence that blood-based DNAm is associated with lifetime cannabis use.

genomics↗