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Biology subjects

Goldys, E.

Publications and source records attributed to Goldys, E..

9 recordsLinked to original sources

A light-responsive lipid nanoparticle platform delivering Cas9 RNP for VEGFA editing suppresses choroidal neovascularisation

Choroidal neovascularisation (CNV) is a major cause of irreversible vision loss. Conventional anti-VEGF therapies for CNV often require repeated intravitreal injections, imposing substantial treatment burden and cumulative injection-related risk. Genome editing to disrupt VEGFA could provide durable suppression, potentially from a single administration. However, durability depends on permanently disrupting the target in a well-controlled way, as unrestricted VEGFA loss can cause choriocapillaris atrophy and compromise retinal function. This represents a major safety barrier to clinical translation. In this study, we developed lipid-based nanoparticles to release Cas9/sgRNA ribonucleoprotein (RNP) complexes under light illumination, enabling light-controlled editing of the VEGFA gene. The nanoparticles had a mean hydrodynamic diameter of approximately 130 nm with low polydispersity (0.296) and high sgRNA encapsulation efficiency (90.33%). In human retinal pigment epithelial cells, light exposure increased the extent of VEGFA disruption relative to the non-illuminated condition, as confirmed by targeted next-generation sequencing, with corresponding reductions in VEGFA mRNA and protein expression. Following a single intravitreal injection in a laser-induced rat CNV model, the nanoparticles combined with light illumination significantly reduced vascular leakage and CNV-associated structural abnormalities compared with both vehicle and non-illuminated controls, and ocular safety assessment revealed no apparent nanoparticle-related structural abnormalities in the retina. This study provides a foundation for spatially controlled VEGFA genome editing as a potential therapeutic strategy for VEGFA-driven macular neovascular disease.

bioengineering↗

Modular split-trigger based cooperative activation platform for Cas13-12 tandem biosensing system

Achieving precise control of CRISPR/Cas trans-cleavage depends on understanding how nucleic acid activators engage Cas effectors, yet the fundamental principles of split-trigger activation of Cas12a remain unclear. Here, we uncover the mechanistic determinants that enable fragmented nucleic acids to collectively initiate Cas12a activity. We show that split triggers bearing external extensions fully support the R-loop formation, whereas internal extensions which disrupt the spacer complementarity abolish Csa12a activation. We further demonstrate that covalent linkage of split-trigger fragments prevents R-loop propagation, revealing that Cas12as activation strictly requires two physically independent split fragments. Together, these findings establish a synergistic split-trigger activation mechanism in which cooperative hybridization of two individually fragments nucleates and extends the Cas12a R-loop with high efficiency. Conceptually, this mechanism enables a cascade architecture that transforms CRISPR diagnostics from a one-target one-Cas ribonucleoprotein (RNP) paradigm into a highly amplifying process in which a single target molecule activates numerous downstream Cas RNPs. Building on this principle, we show that the cleavage of a rationally designed linear DNA-RNA-DNA mediator by LbuCas13a generates optimally configured split triggers for Cas12a activation, thereby coupling RNA recognition to large-scale Cas12a activation without enzymatic preamplification. The resulting Split Trigger Activated Cas13-Cas12 Cascade System (STACS) achieves amplification-free detection down to 1 copy/{micro}L within 15 minutes and maintains robust performance in complex biological (serum, saliva) and environmental (mud) matrices. This work establishes a generalizable strategy for engineering programmable CRISPR cascades with high Cas RNP activation multiplicity for ultrasensitive molecular diagnostics. Graphic abstract O_FIG O_LINKSMALLFIG WIDTH=200 HEIGHT=102 SRC="FIGDIR/small/734747v2_ufig1.gif" ALT="Figure 1"> View larger version (18K): org.highwire.dtl.DTLVardef@104a609org.highwire.dtl.DTLVardef@30cebcorg.highwire.dtl.DTLVardef@54b15borg.highwire.dtl.DTLVardef@47f89e_HPS_FORMAT_FIGEXP M_FIG C_FIG Mechanism and detection workflow of the Split Trigger Activated Cas13-12 Cascade System (STACS).

Molecular Biology↗

Modulating Cas13a trans-cleavage by double strand RNA: Application to the development of an autocatalytic sensor

Cas13a-based diagnostic systems have been widely utilized for the detection of RNA targets. However, without preamplification such systems have sensitivity in the picomolar range only. Here, we found that double strand RNA (dsRNA) over 20nt is able to effectively activate the trans-cleavage activity of Cas13a RNP, while the cleavage rates of dsRNA by activated Cas13a RNP are very low. In addition, specially designed small circular RNA constructs (Cir-mediators comprising a 20nt dsRNA trigger with a 5nt ssRNA linker) have limited ability to activate Cas13a RNP, but this activation is restored once the circular structures are cleaved and become linear. Based on this new method to control trans-cleavage activity of Cas13 RNP, we developed a Cas13a autocatalytic biosensing system assisted by Cir-mediators, which allows one target RNA to activate numerous Cas13a RNPs. With this approach we show ultrasensitive detection of 1aM of synthetic RNA targets without preamplification within 15min. The sensor was successfully applied to monitor miRNA-21 concentration in clinical plasma samples in colorectal cancer. This investigation yields novel insights into the properties of Cas13a RNPs, and the Cir-mediator-based autosensor introduces a novel method for detecting RNA targets with exceptional sensitivity. HighlightsO_LIdsRNA is able to trigger activation of Cas13a RNP. C_LIO_LIActivated Cas13a RNP do not cleave dsRNA. C_LIO_LICir-mediator induces low levels of Cas13a RNP activation. C_LIO_LICir-mediator based Cas13a auto-catalysis biosensor can detect 1aM RNA targets. C_LI

molecular biology↗

Integration of hyperspectral imaging and transcriptomics from individual cells with HyperSeq

Microscopy and omics are complementary approaches to probe the molecular state of cells in health and disease, combining granularity with scalability. While important advances have been achieved over the last decade in each area, integrating both imaging- and sequencing-based assays on the same cell has proven challenging. In this study, a new approach called HyperSeq that combines hyperspectral autofluorescence imaging with transcriptomics on the same cell is demonstrated. HyperSeq was applied to Michigan Cancer Foundation 7 (MCF-7) breast cancer cells and identified a subpopulation of cells exhibiting bright autofluorescence rings at the plasma membrane in optical channel 13 ({lambda}ex = 431 nm,{lambda} em = 594 nm). Correlating the presence of a ring with the gene expression in the same cell indicated that ringed cells are more likely to express hallmark genes of apoptosis and less likely to express genes associated with ATP production. Further, correlation of cell morphology with gene expression suggested that multiple members of the spliceosome were downregulated in larger MCF-7 cells. Multiple genes were evenly expressed across cell sizes but also exhibited higher usage of specific exons in larger or smaller cells. Finally, correlation between gene expression and fluorescence within the spectral range of Nicotinamide adenine dinucleotide hydrogen (NADH) provided insight into the metabolic states of MCF-7 cells. These observations provided a link between the cells optical spectrum and its internal molecular state, demonstrating the utility of HyperSeq to study cell biology at single cell resolution by integrating spectral, morphological and transcriptomic analyses into a single, streamlined workflow.

bioinformatics↗

Automated identification of aneuploid cells within the inner cell mass of an embryo using a numerical extraction of morphological signatures

STUDY QUESTIONCan artificial intelligence distinguish between euploid and aneuploid cells within the inner cell mass of mouse embryos using brightfield images? SUMMARY ANSWERA deep morphological signature (DMS) generated by deep learning followed by swarm intelligence and discriminative analysis can identify the ploidy state of inner cell mass (ICM) in the mouse blastocyst-stage embryo. WHAT IS KNOWN ALREADYThe presence of aneuploidy - a deviation from the expected number of chromosomes - is predicted to cause early pregnancy loss or congenital disorders. To date, available techniques to detect embryo aneuploidy in IVF clinics involve an invasive biopsy of trophectoderm cells or a non-invasive analysis of cell-free DNA from spent media. These approaches, however, are not specific to the ICM and will consequently not always give an accurate indication of the presence of aneuploid cells with known ploidy therein. STUDY DESIGN, SIZE, DURATIONThe effect of aneuploidy on the morphology of ICMs from mouse embryos was studied using images taken using a standard brightfield microscope. Aneuploidy was induced using the spindle assembly checkpoint inhibitor, reversine (n = 13 euploid and n = 9 aneuploid). The morphology of primary human fibroblast cells with known ploidy was also assessed. PARTICIPANTS/MATERIALS, SETTING, METHODSTwo models were applied to investigate whether the morphological details captured by brightfield microscopy could be used to identify aneuploidy. First, primary human fibroblasts with known karyotypes (two euploid and trisomy: 21, 18, 13, 15, 22, XXX and XXY) were imaged. An advanced methodology of deep learning followed by swarm intelligence and discriminative analysis was used to train a deep morphological signature (DMS). Testing of the DMS demonstrated that there are common cellular features across different forms of aneuploidy detectable by this approach. Second, the same approach was applied to ICM images from control and reversine treated embryos. Karyotype of ICMs was confirmed by mechanical dissection and whole genome sequencing. MAIN RESULTS AND THE ROLE OF CHANCEThe DMS for discriminating euploid and aneuploid fibroblasts had an area under the receiver operator characteristic curve (AUC-ROC) of 0.89. The presence of aneuploidy also had a strong impact on ICM morphology (AUC-ROC = 0.98). Aneuploid fibroblasts treated with reversine and projected onto the DMS space mapped with untreated aneuploid fibroblasts, supported that the DMS is sensitive to aneuploidy in the ICMs, and not a non-specific effect of the reversine treatment. Consistent findings in different contexts suggests that the role of chance low. LARGE SCALE DATAN/A LIMITATIONS, REASON FOR CAUTIONConfirmation of this approach in humans is necessary for translation. WIDER IMPLICATIONS OF THE FINDINGSThe application of deep learning followed by swarm intelligence and discriminative analysis for the development of a DMS to detect euploidy and aneuploidy in the ICM has high potential for clinical implementation as the only equipment it requires is a brightfield microscope, which are already present in any embryology laboratory. This makes it a low cost, a non-invasive approach compared to other types of pre-implantation genetic testing for aneuploidy. This study gives proof of concept for a novel strategy with the potential to enhance the treatment efficacy and prognosis capability for infertility patients. STUDY FUNDING/COMPETING INTEREST(S)K.R.D. is supported by a Mid-Career Fellowship from the Hospital Research Foundation (C-MCF-58-2019). This study was funded by the Australian Research Council Centre of Excellence for Nanoscale Biophotonics (CE140100003), the National Health and Medical Research Council (APP2003786) and an ARC Discovery Project (DP210102960). The authors declare that there is no conflict of interest.

bioinformatics↗

Oocyte and cumulus cell cooperativity and metabolic plasticity under the direction of oocyte paracrine factors

Mammalian oocytes develop and mature in a mutually dependent relationship with surrounding cumulus cells. The oocyte actively regulates cumulus cell differentiation and function by secreting soluble paracrine oocyte-secreted factors (OSFs). We characterized the molecular mechanisms by which two model OSFs, cumulin and BMP15, regulate oocyte maturation and cumulus-oocyte cooperativity. Exposure to these OSFs during maturation altered the proteomic and multispectral autofluorescence profiles of both the oocyte and cumulus cells. In oocytes, cumulin significantly upregulated proteins involved in nuclear function. In cumulus cells, both OSFs elicited marked upregulation of a variety of metabolic processes (mostly anabolic), including lipid, nucleotide, and carbohydrate metabolism, while mitochondrial metabolic processes were downregulated. The mitochondrial changes were validated by functional assays confirming altered mitochondrial morphology, respiration, and content, whilst maintaining ATP homeostasis. Collectively, these data demonstrate that OSFs remodel cumulus cell metabolism during oocyte maturation in preparation for ensuing fertilization and embryonic development. HIGHLIGHTSO_LIDuring oocyte maturation, oocyte-secreted factors promote cell cooperativity between the oocyte and cumulus cells by altering the molecular composition of both cell types. C_LIO_LIOocyte-secreted factors downregulate protein catabolic processes, and upregulate DNA binding, translation, and ribosome assembly in oocytes. C_LIO_LIOocyte-secreted factors alter mitochondrial number, morphology, and function in cumulus cells. C_LIO_LIOocyte-secreted factors further enhance metabolic plasticity in cumulus cells by upregulating anabolic pathways for macromolecules and small molecule organics. C_LIO_LIThe oocyte, via oocyte-secreted factors, instructs cumulus cells to increase metabolic workload on its behalf, thereby subduing oocyte metabolism. C_LI

developmental biology↗

moRphology - dEep Learning Imaging Cells (RELIC) - to Differentiate Between Normal and Pathological Kidney Exfoliated Cells

Chronic kidney disease (CKD) is characterised by progressive loss of kidney function leading to kidney failure. Significant kidney damage can occur before symptoms are detected. Currently, kidney tissue biopsy is the gold standard for evaluation of renal damage and CKD severity. This study explores how to precisely quantify morphology characteristics of kidney cells exfoliated into urine, with a view to establish a future urine-based non-invasive diagnostic for CKD. We report the development of a novel deep learning method, which was able to discover a RELIC (moRphology dEep Learning Imaging Cells) signature that can differentiate between kidney cells exfoliated in human urine of and CKD patients with varying degree of kidney damage and non-CKD controls. Exfoliated proximal tubule cells (PTCs) originating from kidneys were isolated from the urine of patients with different levels of kidney damage using previously published methods. An advanced combination of artificial intelligence techniques, deep learning, swarm intelligence, and discriminative analysis was used to discover a RELIC signature in brightfield microscopy images of exfoliated PTCs. Kidney damage in the study subjects was characterised by assessing kidney tissues obtained through a nephrectomy or kidney biopsy. A special deep learning algorithm was developed and trained to create a predictive tool. The algorithm was then used to analyse data from patients with normal and fibrotic kidneys. Data were then classified according to different groups (healthy or fibrosis) and clustering of the training and validation cells was determined for model validation. We developed a novel deep learning method, to obtain RELIC signatures and identify specific deep morphological features which can be used to differentiate urinary PTC cells shed by people with CKD (confirmed by tissue histology obtained from an invasive kidney biopsy) from those without CKD, with a discriminatory accuracy of 82%. We identified a RELIC signature which can be used on a collection of bright field images of exfoliated urinary PTCs to create a predictive tool and differentiate between normal and pathological kidney cells. This study, for the first time, provides a proof of concept that urinary exfoliated tubule cells in patients with kidney fibrosis and healthy controls differ in appearance (morphology) as observed under a basic brightfield microscope. The results suggest that morphological signatures of exfoliated PTCs have the potential to serve as a non-invasive marker of kidney fibrosis.

bioinformatics↗

Non-invasive real-time imaging of reactive oxygen species (ROS) using multispectral auto-fluorescence imaging technique: a novel tool for redox biology

Detecting reactive oxygen species (ROS) that play a critical role as redox modulators and signalling molecules in biological systems currently requires invasive methods such as ROS - specific indicators for imaging and quantification. We developed a non-invasive, real-time, label-free imaging technique for assessing the level of ROS in live cells and thawed cryopreserved tissues that is compatible with in-vivo imaging. The technique is based on autofluorescence multispectral imaging (AFMI) carried out in an adapted fluorescence microscope with an expanded number of spectral channels spanning specific excitation (365 nm-495 nm) and emission (420 nm-700 nm) wavelength ranges. We established a strong quantitative correlation between the spectral information obtained from AFMI and the level of ROS obtained from CellROX staining. The results were obtained in several cell types (HeLa, PANC1 and mesenchymal stem cells) and in live kidney tissue. Additioanly, two spectral regimes were considered: with and without UV excitation (wavelengths > 400 nm); the latter being suitable for UV-sensitive systems such as the eye. Data were analyzed by linear regression combined with an optimization method of swarm intelligence. This allowed the calibration of AFMI signals to the level of ROS with excellent correlation (R= 0.84, p=0.00) in the entire spectral range and very good correlation (R= 0.78, p=0.00) in the limited, UV-free spectral range. We also developed a strong classifier which allowed us to distinguish moderate and high levels of ROS in these two regimes (AUC= 0.91 in the entire spectral range and AUC = 0.78 for UV-free imaging). These results indicate that ROS in cells and tissues can be imaged non-invasively, which opens the way to future clinical applications in conditions where reactive oxygen species are known to contribute to progressive disease such as in ophthalmology, diabetes, kidney disease, cancer and neurodegenerative diseases.

cell biology↗

NAD+ repletion rescues female fertility during reproductive ageing

Female infertility is a common and devastating condition with life-long health, emotional and social consequences. There is currently no pharmacological therapy for preserving oocyte quality during aging, which is the strongest risk factor for infertility. This leads to an age dependent decline in natural conception and IVF success rates (1). Here, we show that this is due in part to declining levels of the metabolic cofactor nicotinamide adenine dinucleotide (NAD+), and that restoring NAD+ levels with its metabolic precursor nicotinamide mononucleotide (NMN) rejuvenates oocyte quality and quantity in aged animals, leading to improved fertility. These benefits extend to the developing embryo, where NMN supplementation in embryo culture media following IVF enhances blastocyst formation in older mice. The NAD+ dependent deacylase SIRT2 is sufficient, but not essential, to recapitulate the benefits of in vivo NMN treatment, and transgenic overexpression of SIRT2 maintains oocyte spindle assembly, accurate chromosome segregation, decreased oxidative stress and overall fertility with ageing. Pharmacological elevation of NAD+ may be an effective, non-invasive strategy for restoring and maintaining female fertility during ageing, and for improving the success of IVF.

developmental biology↗