Priya Karmali
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View article: 1202 In vivo engineering of CAR T cells using a novel targeted LNP-mRNA technology
1202 In vivo engineering of CAR T cells using a novel targeted LNP-mRNA technology Open
Background Autologous chimeric antigen receptor (CAR) T cell therapies have revolutionized the treatment of some cancers and are now demonstrating effects in autoimmune disease. With several approved therapies on the market and cure rates …
View article: Engineered tRNAs suppress nonsense mutations in cells and in vivo
Engineered tRNAs suppress nonsense mutations in cells and in vivo Open
Nonsense mutations are the underlying cause of approximately 11% of all inherited genetic diseases 1 . Nonsense mutations convert a sense codon that is decoded by tRNA into a premature termination codon (PTC), resulting in an abrupt termin…
View article: Supplementary Figure 2S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 2S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Expression levels of miR-17 TuD. The data are expressed as delta CT value normalized to GAPDH.
View article: Supplementary Figure 3S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 3S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Inhibition of miR-17 family by miR-17 TuD had no effect on caspase 3/7 activity. Caspase 3/7 activity was represented as relative activity compared to NC TuD expression cells.
View article: Supplementary Methods and legends from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Methods and legends from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Supplementary Methods and Figure legends
View article: Data from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Data from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Hepatocellular carcinoma (HCC) is one of the most common human malignancies with poor prognosis and urgent unmet medical need. Aberrant expression of multiple members of the miR-17 family are frequently observed in HCC, and their overexpre…
View article: Supplementary Methods and legends from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Methods and legends from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Supplementary Methods and Figure legends
View article: Supplementary Figure 4S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 4S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
MetaCoreTM schematic of the TGF-β signaling pathway showing genes regulated by miR-17 TuD in Hep3B (1) and HepG2 (2).
View article: Supplementary Figure 2S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 2S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Expression levels of miR-17 TuD. The data are expressed as delta CT value normalized to GAPDH.
View article: Supplementary Figure 1S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 1S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Inhibition of miR-17, 20a, 20b, 93 and 106b activity by miR-17 TuD in HeLa cells as determined by luciferase reporter assays.
View article: Data from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Data from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Hepatocellular carcinoma (HCC) is one of the most common human malignancies with poor prognosis and urgent unmet medical need. Aberrant expression of multiple members of the miR-17 family are frequently observed in HCC, and their overexpre…
View article: Supplementary Figure 3S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 3S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Inhibition of miR-17 family by miR-17 TuD had no effect on caspase 3/7 activity. Caspase 3/7 activity was represented as relative activity compared to NC TuD expression cells.
View article: Supplementary Figure 1S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 1S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
Inhibition of miR-17, 20a, 20b, 93 and 106b activity by miR-17 TuD in HeLa cells as determined by luciferase reporter assays.
View article: Supplementary Figure 4S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth
Supplementary Figure 4S from Lipid Nanoparticle–Mediated Delivery of Anti-miR-17 Family Oligonucleotide Suppresses Hepatocellular Carcinoma Growth Open
MetaCoreTM schematic of the TGF-β signaling pathway showing genes regulated by miR-17 TuD in Hep3B (1) and HepG2 (2).
View article: Liver lipopolysaccharide binding protein prevents hepatic inflammation in physiological and pathological non-obesogenic conditions
Liver lipopolysaccharide binding protein prevents hepatic inflammation in physiological and pathological non-obesogenic conditions Open
Lipopolysaccharide binding protein (LBP) knockout mice models are protected against the deleterious effects of major acute inflammation but its possible physiological role has been less well studied. We aimed to evaluate the impact of live…
View article: Downregulation of hepatic lipopolysaccharide binding protein improves lipogenesis-induced liver lipid accumulation
Downregulation of hepatic lipopolysaccharide binding protein improves lipogenesis-induced liver lipid accumulation Open
View article: Downregulation of peripheral lipopolysaccharide binding protein impacts on perigonadal adipose tissue only in female mice
Downregulation of peripheral lipopolysaccharide binding protein impacts on perigonadal adipose tissue only in female mice Open
LBP seems to exert a negative feedback on ERα-mediated estrogen action, impacting on genes involved in thermogenesis. The known decreased estrogen action and negative effects of metabolic endotoxemia may be targeted through LBP downregulat…
View article: Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model
Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model Open
Phenylketonuria (PKU) is a genetic disorder affecting around 1 in 12,000 live births (1), caused by a mutation in the phenylalanine hydroxylase (PAH) gene in the liver which facilitates the catabolism of phenylalanine (Phe). Without a func…
View article: Hepatocyte-specific activity of TSC22D4 triggers progressive NAFLD by impairing mitochondrial function
Hepatocyte-specific activity of TSC22D4 triggers progressive NAFLD by impairing mitochondrial function Open
View article: Development of an mRNA replacement therapy for phenylketonuria
Development of an mRNA replacement therapy for phenylketonuria Open
View article: Synthesis and bioactivity of readily hydrolysable novel cationic lipids for potential lung delivery application of mRNAs
Synthesis and bioactivity of readily hydrolysable novel cationic lipids for potential lung delivery application of mRNAs Open
View article: Hepatocyte-specific activity of TSC22D4 triggers progressive NAFLD by impairing mitochondrial function
Hepatocyte-specific activity of TSC22D4 triggers progressive NAFLD by impairing mitochondrial function Open
Objective Fibrotic organ responses have recently been identified as long-term complication in diabetes. Indeed, insulin resistance and aberrant hepatic lipid accumulation represent driving features of progressive non-alcoholic fatty liver …
View article: 549: LUNAR-CF: A novel mRNA therapeutic approach to correct the root cause of cystic fibrosis lung disease
549: LUNAR-CF: A novel mRNA therapeutic approach to correct the root cause of cystic fibrosis lung disease Open
View article: A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice
A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice Open
View article: Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS
Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS Open
View article: CD8 <sup>+</sup> T cells mediate protection against Zika virus induced by an NS3-based vaccine
CD8 <sup>+</sup> T cells mediate protection against Zika virus induced by an NS3-based vaccine Open
The crucial role of CD8 + T cells in vaccine-mediated protection is a valuable evidence for new insights in vaccine development.
View article: A Single Dose of Self-Transcribing and Replicating RNA Based SARS-CoV-2 Vaccine Produces Protective Adaptive Immunity In Mice
A Single Dose of Self-Transcribing and Replicating RNA Based SARS-CoV-2 Vaccine Produces Protective Adaptive Immunity In Mice Open
A self-transcribing and replicating RNA (STARR™) based vaccine (LUNAR ® -COV19) has been developed to prevent SARS-CoV-2 infection. The vaccine encodes an alphavirus-based replicon and the SARS-CoV-2 full length spike glycoprotein. Transla…
View article: Lipid Nanoparticle Formulation Increases Efficiency of DNA-Vectored Vaccines/Immunoprophylaxis in Animals Including Transchromosomic Bovines
Lipid Nanoparticle Formulation Increases Efficiency of DNA-Vectored Vaccines/Immunoprophylaxis in Animals Including Transchromosomic Bovines Open
The use of nucleic acid as a drug substance for vaccines and other gene-based medicines continues to evolve. Here, we have used a technology originally developed for mRNA in vivo delivery to enhance the immunogenicity of DNA vaccines. We d…
View article: Anti-HFRS Human IgG Produced in Transchromosomic Bovines Has Potent Hantavirus Neutralizing Activity and Is Protective in Animal Models
Anti-HFRS Human IgG Produced in Transchromosomic Bovines Has Potent Hantavirus Neutralizing Activity and Is Protective in Animal Models Open
We explored an emerging technology to produce anti-Hantaan virus (HTNV) and anti-Puumala virus (PUUV) neutralizing antibodies for use as pre- or post-exposure prophylactics. The technology involves hyperimmunization of transchomosomic bovi…
View article: Anti-miR-17 therapy delays tumorigenesis in MYC-driven hepatocellular carcinoma (HCC)
Anti-miR-17 therapy delays tumorigenesis in MYC-driven hepatocellular carcinoma (HCC) Open
Hepatocellular carcinoma (HCC) remains a significant clinical challenge with few therapeutic options. Genomic amplification and/or overexpression of the MYC oncogene is a common molecular event in HCC, thus making it an attractive target f…