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Custom-designed lipid-based nanoparticles efficiently deliver mRNA to reverse the pathogenesis of LAL deficiency in a preclinical model

ABSTRACT: Custom-designed lipid-based nanoparticles efficiently deliver mRNA to reverse the pathogenesis of LAL deficiency in a preclinical model

PROVIDER: PRJNA1181265 | ENA |

REPOSITORIES: ENA

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			Action	DRS
	SRR31203118.fastq.gz	Fastqsanger.gz
	SRR31203119.fastq.gz	Fastqsanger.gz
	SRR31203120.fastq.gz	Fastqsanger.gz
	SRR31203121.fastq.gz	Fastqsanger.gz
	SRR31203122.fastq.gz	Fastqsanger.gz

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Custom-designed lipid-based nanoparticles efficiently deliver mRNA to reverse the pathogenesis of LAL deficiency in a preclinical model

Project description:Lysosomal acid lipase (LAL) is a crucial enzyme responsible for the hydrolysis of triglycerides (TGs) and cholesteryl esters (CEs) in the lysosomes. LAL deficiency results in the extensive storage of CEs and TGs in the liver and the spleen, inducing hepatosplenomegaly, and can be a life-threatening condition with patients rarely surviving beyond 6 months of age in the most aggressive form. Using messenger ribonucleic acid (mRNA) for protein replacement is an innovative approach to treating LAL deficiency. Here, we describe the development of a new lipid-based nanoparticle (LNP) formulation capable of efficiently delivering LAL mRNA and restoring LAL activity in affected organs mediating significant reversal of the pathological progression in a highly predictive preclinical model. A combinatorial library of mRNA-LNPs was generated and screened both in vitro and in vivo to yield a new formulation with a higher potency than an FDA-approved MC3-based nano-formulation. In vivo evaluation revealed that the new formulation could promote a more sustained and superior LAL expression. Multiple injection regimen was able to mitigate hepatosplenomegaly and reduce the lipid accumulation of CEs and TGs by 20-30% in the liver and 50% in the spleen. Furthermore, liver inflammation processes and development of fibrosis were significantly diminished. These findings provide strong evidence that mRNA-LNP is a very promising approach for the chronic treatment of LAL deficiency and support the clinical translation of mRNA therapy to overcome side effects and challenges encountered with traditional enzyme replacement therapies.

2024-12-06 | GSE280933 | GEO

Copy number alteration in Wilms' tumor with custom-designed miRNA probes

Project description:The aim of this study is to discover loss of specific miRNA loci in Wilms' tumors using array CGH. Custom arrays were designed based on the Agilent 2x105K Human Whole Genome Genomic microarray with Agilent’s eArray program (https://earray.chem.agilent.com/earray/), with additional probes that cover all miRNA regions (200 bps before, within and after each miRNA from miRBase v13, with each probe in triplicates to enhance the reliability). All custom-designed probes were designed in UCSC hg18. Probes from Agilent’s database were lifted-over from hg19 to hg18 (LiftOver tool: http://genome.ucsc.edu).

2012-03-31 | GSE28397 | GEO

Calibration of a custom designed microarray

2012-03-27 | GSE36800 | GEO

Effect of knockdEffect of knockdown of mitochondrial atp1 mRNA by a custom-designed PPR protein on protein expression in Arabidopsis thaliana own of mitochondrial atp1 mRNA by a custom-designed PPR protein on protein expression in Arabidopsis thaliana

Project description:We show that a custom-designed RNA-binding protein binds and specifically induces cleavage of atp1 RNA in mitochondria, significantly decreasing the abundance of the Atp1 protein and the assembled F1Fo ATP synthase in Arabidopsis thaliana. Changes to the proteome as a consequence of the modification were investigated by shotgun proteomics on enriched mitochondrial fractions.

2023-10-24 | PXD037659 | Pride

Copy number alteration in Wilms' tumor with custom-designed miRNA probes

2012-03-30 | E-GEOD-28397 | biostudies-arrayexpress

Calibration of a custom designed microarray for European Fire Salamander

Project description:A custom 8x60 k expression microarray for larvae of European fire salamander (Salamandra salamandra) was designed based on transcriptome sequencing. It is known the fact, that oligonucleotide probes differ in the binding behavior towards their target sequences. Therefore, we performed a calibration of our microarray where we assessed the binding behavior of the individual probes empirically. This information was used to normalize gene expression data measurements with the same microarray in another experiment. Please refer to the accompanying publication (Czypionka et al. 2015." Ecological transcriptomics – a non-lethal sampling approach for endangered fire salamanders" Methods in Ecology and Evolution) for more information.

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custom mNGS

Project description:custom mNGS

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Calibration of a custom designed microarray

Project description:We designed a custom expression 8x15 k microarray for Cottus fishes based on transcriptome sequencing. It is a known fact, that oligonucleotide probes differ in the binding behavior towards their target sequences. Therefore, we performed a calibration of our microarray where we assessed the binding behavior of the individual probes empirically. This information was used to normalize gene expression data measurements with the same microarray in another experiment. Please refer to the accompanying publication (Czypionka et al. 2011."Transcriptome changes after genome wide admixture in invasive sculpins" Molecular Ecology; no doi yet) for more information. Labeled cRNA was prepared from 3 Cottus fish. These 3 fish were representative of species (C.rhenanus, C.perifretum, invasive hybrid species) used in another experiment. An calibration pool was prepared from equimolar amounts of this cRNA. Increasing amounts of labeled cRNA (75 ng, 150 ng, 300 ng, 600 ng, 1200 ng, 2400 ng, 4800 ng), corresponding to (1/8, 1/4, 1/2, 1, 2, 4 and 8 times the recommended amount of 600 ng) were hybridized to 7 microarrays (one microarray per dilution-step). The change in observed signal intensity in relation to the change in amount of labeled cRNA was used to infer the target-binding behavior of the individual probes. This information was extracted, to be used for a normalization procedure in another experiment with the same microarray (see Czypionka et al. 2011."Transcriptome changes after genome wide admixture in invasive sculpins" Molecular Ecology; no doi yet)

2012-03-26 | E-GEOD-36800 | biostudies-arrayexpress

Lipid nanoparticles deliver DNA-encoded biologics and induce potent protective immunity

Project description:Lipid nanoparticles deliver DNA-encoded biologics and induce potent protective immunity

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Clay nanoparticles efficiently deliver small interfering RNA to intact plant leaf cells.

Project description:RNA interference is triggered in plants by the exogenous application of double-stranded RNA or small interfering RNA (siRNA) to silence the expression of target genes. This approach can potentially provide insights into metabolic pathways and gene function and afford plant protection against viruses and other plant pathogens. However, the effective delivery of biomolecules such as siRNA into plant cells is difficult because of the unique barrier imposed by the plant cell wall. Here, we demonstrate that 40-nm layered double hydroxide (LDH) nanoparticles are rapidly taken up by intact Nicotiana benthamiana leaf cells and by chloroplasts, following their application via infiltration. We also describe the distribution of infiltrated LDH nanoparticles in leaves and demonstrate their translocation through the apoplast and vasculature system. Furthermore, we show that 40-nm LDH nanoparticles can greatly enhance the internalization of nucleic acids by N. benthamiana leaf cells to facilitate siRNA-mediated downregulation of targeted transgene mRNA by >70% within 1 day of exogenous application. Together, our results show that 40-nm LDH nanoparticle is an effective platform for delivery of siRNA into intact plant leaf cells.

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