Camargo

JULY 27, 2021

It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy. Additionally, gene editing allows us either to remove or to modify harmful genes. Formulation Considerations.

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Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Drugs Gene Editing Genome Genomics 52

Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.

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Gene Expression Gene DNA RNA 52

Scienmag

NOVEMBER 26, 2020

For the first time, researchers describe how Rho protein really stops gene expression COLUMBUS, Ohio – New research has identified and described a cellular process that, despite what textbooks say, has remained elusive to scientists until now – precisely how the copying of genetic material that, once started, is properly turned off.

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Gene Expression Genetics Protein Gene 54

Scienmag

SEPTEMBER 28, 2020

Brazilian research group shows that valproic acid (VPA), used to treat epilepsy since the 1960s, modulates gene expression in tumor gene models and acts on DNA conformation and the histones in chromatin Results of recent studies involving valproic acid, used for decades as an anti-convulsant drug, show that it can interact with the conformation of (..)

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DNA Gene Expression Protein Gene 70

Scienmag

SEPTEMBER 10, 2020

Findings suggest one factor that may contribute to disparities in COVID-19 infection New York – In a study published in JAMA today, Mount Sinai researchers report findings that shed some light on the disproportionate impact of COVID-19 on Blacks, who have experienced rates of infection and death that are much greater, in some areas twice […]. (..)

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Gene Expression Protein Gene Research 40

pharmaphorum

NOVEMBER 12, 2021

By tying these to underlying disease-protein-pathway relationships, the AI was also able to suggest a list of drugs – many of them untested in COVID-19 – that could be candidates for treatments of these complications.

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Doctors Doctor Drugs Gene Expression 135

Drug Discovery World

SEPTEMBER 27, 2023

Embarking on a new era of medicine The advent of genomics has ushered in the era of personalised medicine, enabling us to analyse the genetic makeup of individuals with unprecedented accuracy. Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism.

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DNA Genetics Gene Medicine 115

Scienmag

APRIL 5, 2021

Study shows that tumor-suppressor protein p53 brings speckles and DNA together to boost gene expression PHILADELPHIA – A team led by scientists at the Perelman School of Medicine at the University of Pennsylvania has illuminated the functions of mysterious structures in cells called “nuclear speckles,” showing that they can work in (..)

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Gene Gene Expression DNA Protein 42

Roots Analysis

JANUARY 14, 2024

Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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Contract Manufacturing Manufacturing Protein Bacteria 40

Drug Discovery World

MARCH 21, 2023

In July 2022, DDW reported that molecular genetics company Mainz Biomed had enrolled its first patient in an international clinical study assessing the potential combination of gene expression (mRNA) biomarkers with a diagnostic test for colorectal cancer.

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Drugs Gene Expression RNA Clinical Trials 52

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Visium Spatial Gene Expression Solution. Feature barcoding using gel beads. Single Cell Data Analysis.

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Gene Expression Development RNA Drugs 112

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccination Vaccine 75

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy. Secondly, other funded research is exploring differential gene expression regulated by DNA methylation.

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Gene Therapy Gene Life Science DNA 111

pharmaphorum

MARCH 10, 2021

The US biotech said the suspected serious adverse reaction reported in the phase 1/2 study of LentiGlobin gene therapy for sickle cell disease was unlikely to be related to the virus vector used in the therapy, used to deliver the genetic material to the body. Shares in bluebird ticked up following the announcement.

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Gene Therapy Gene Trials Gene Expression 53

The Pharma Data

JUNE 14, 2023

Abell Chair in Molecular and Cellular Biology – and Pengbo Cao, a postdoctoral researcher in Whiteley’s lab, discovered a gene that drives the switch. By measuring bacterial gene expression in human tissue samples, the researchers identified a biomarker for the transition.

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RNA Regulation Scientist Bacteria 40

pharmaphorum

JANUARY 29, 2021

ZFPs can be engineered to make zinc finger nucleases, or ZFNs, which are proteins that can be used to edit genomes by knocking select genes in or out to specifically modify DNA sequences. Zinc fingers are the most common control gene in the body,” Macrae explains. First of all it will be used for ultra-rare monogenic disease.

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Gene Editing Gene Gene Therapy In-Vivo 145

XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics. Rosalind Franklin.

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Genetics DNA Genome Genomics 119

Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate.

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Gene Gene Therapy Gene Expression Regulation 40

Pharmaceutical Technology

JUNE 13, 2023

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression.

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Gene Expression Protein Sales Genetics 130

Drug Discovery World

DECEMBER 12, 2022

According to the publication, “A small clinical trial has shown that researchers can use CRISPR gene editing to alter immune cells so that they will recognise mutated proteins specific to a person’s tumours. In Nature 3 , we learnt about the CRISPR cancer trial success leading the way to personalised treatments.

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Drugs Gene Editing Genome Genomics 52

XTalks

MARCH 22, 2021

Using a simple, standard blood sample, the assay can detect actionable genetic mutations in cancers, including cancer-driving alterations. Maverick’s COBRA platform is used to engineer protein-based therapies that target T cells in the tumor microenvironment to trigger their activation.

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Life Science Development Marketing Gene Expression 110

Roots Analysis

AUGUST 31, 2023

At present, extensive studies are being conducted to understand the interaction between nutrients and gene expression at the molecular level, to determine the effect of a specific nutrient and dietary regime on human health. It is worth mentioning that more than 50 genes in a human body are associated with obesity.

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Genome Genomics Genetics Gene 40

XTalks

MARCH 7, 2024

Breast Cancer Multiomics: Unified Insights in Tumor Heterogeneity This on-demand webinar introduces a cutting-edge single-cell multiomics method designed for garnering detailed genetic insights into breast cancer. The test accurately identified a range of genetic abnormalities, including trisomies and microdeletions.

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Hormones Genetics Genome Genomics 115

Drug Discovery World

APRIL 14, 2023

DDW Editor Reece Armstrong speaks to Nautilus Co-Founder and Chief Scientist Parag Mallick , about the company’s recent collaboration which aims to uncover the proteins that could be behind a rare and fatal childhood cancer. To do this, Nautilus will useits protein analysis platform to provide TGen with data on the proteins involved in DPIG.

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Protein Genome Genomics DNA 52

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

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RNA Protein Genetics Gene Expression 40

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

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DNA Gene Gene Silencing Genome 98

XTalks

SEPTEMBER 21, 2020

A study from the University of California San Francisco (UCSF) has revealed that women with Alzheimer’s do better than men with the disease due to a genetic advantage conferred by their extra X chromosome. The gene, called KDM6A , is a histone demethylase that is believed to function as a tumor suppressor. KDM6A Variant.

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Gene Protein Gene Expression Scientist 89

Drug Discovery World

NOVEMBER 15, 2022

First off, genetically encoding desired features into living cells requires designing an appropriate vector, successfully transfecting it across the cell membrane, integrating the new DNA into the host’s genome, and finally, a reporting marker to examine changes. “The This approach has been around for a while.

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DNA Drugs Antibody Engineer 98

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation.

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Marketing Gene Drugs Protein 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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DNA Gene Gene Silencing Genome 52

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Likewise, Lessard et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

APRIL 21, 2023

SLAS Student Poster Award The SLAS Student Poster Award recognises innovative research by students, graduate students, post- doctoral associates and junior faculty (less than four years in first academic appointment) who are chosen to present a poster during SLAS Europe 2023.

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Doctors Doctor Genetics In-Vitro 52

Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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DNA Genome Genomics RNA 98

Drug Discovery World

FEBRUARY 6, 2023

A case in point is the emerging omics and other high-throughput laboratory technologies that allow simultaneous profiling of genetic alterations in combination with profiling thousands of molecules in plasma, cell populations, or single cells. This could be expanded to assessing predictors at baseline.

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Big Data RNA Bioinformatics Vaccination 52

The Pharma Data

DECEMBER 27, 2020

The drug is a topical ointment applied to the lower lid to address the build-up and shedding of proteins at the opening of the Meibomian gland. GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder and a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases. Most Read Today.

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Trials Antibody Gene Therapy Clinical Trials 52

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Single-cell RNA-sequencing of differentiating iPS cells reveals dynamic genetic effects on gene expression.

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Gene Editing Gene Engineer DNA 52