Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Gene Therapy Gene Gene Editing Genetics 238

Scienmag

DECEMBER 21, 2020

LJI scientists track down control elements that govern gene expression from a distance Credit: Alex Fung LA JOLLA–The COVID-19 pandemic has highlighted how our small genetic differences can have a tremendous effect on how our bodies respond to disease. “The difference is within us,” says Vivek Chandra, Ph.D.,

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Gene Expression Gene Genetics Scientist 52

BioSpace

JULY 11, 2022

Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.

Genetics 102

Genetics Gene Expression Medicine Engineer 102

Scienmag

MARCH 9, 2021

CHOP researchers find small changes in RNA due to RNA editing could have a large impact on gene expression and phenotype Philadelphia, March 9, 2021–Combining computational mining of big data with experimental testing in the lab, researchers at Children’s Hospital of Philadelphia (CHOP) have identified RNA editing events that influence (..)

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Gene Expression RNA Gene Big Data 53

Scienmag

OCTOBER 1, 2020

The type and direction of the force on a cell alters gene expression by stretching different regions of DNA, researchers at University of […]. . — Tissues and cells in the human body are subjected to a constant push and pull – strained by other cells, blood pressure and fluid flow, to name a few.

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Gene Expression Gene DNA Research 70

Drug Discovery Today

APRIL 24, 2023

Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar genetics.

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Gene Gene Expression Genome Genomics 80

Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

Genetics 130

Genetics Gene Expression DNA Antibody 130

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

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Gene Expression Gene Genetic Disease RNA 59

BioTech 365

APRIL 9, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: New, reversible CRISPR method can control gene expression while leaving underlying DNA sequence unchanged.Over the past decade, the CRISPR-Cas9 gene editing system has revolutionized genetic engineering, allowing … Continue (..)

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Gene Expression DNA Gene Editing Gene 40

Medical Xpress

MARCH 8, 2023

Scientific evidence shows how the cognitive decline in Alzheimer's disease (AD) is caused by the buildup of amyloid beta proteins, which promote synaptic malfunction.

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Gene Expression Scientist Gene Protein 84

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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RNA Gene Expression Gene Gene Therapy 245

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. “Our findings paint a complex picture – with many, dynamic factors driving how tumours respond to treatment.”

Genetics 52

Genetics Gene Hormones RNA 52

Drug Discovery World

FEBRUARY 27, 2024

DDW’s Megan Thomas speaks with Dr Blythe Sather, Vice President and Head of Research at Tune Therapeutics, about how genetic tuning will transform the reach for CAR-T therapies, the different diseases that can benefit, and the disruptive technology making this happen. MT: What is genetic tuning?

Gene Expression 59

Gene Expression DNA Genetics Gene 59

Scienmag

JULY 12, 2021

The function of a protein can depend on its abundance in a cell. So, when investigating the properties of a new protein, it is essential to make sure that the same amount is produced by every cell. Researchers at Baylor College of Medicine and Rice University have found a new way to do just that […].

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Gene Expression Engineer Gene Protein 63

pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.

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Gene Editing Gene Gene Therapy In-Vivo 143

Scienmag

DECEMBER 22, 2020

Blocking gene expression in mitochondria in mice stops cancer cells from growing Credit: Hauke S. The new compound prevents the genetic information within mitochondria from being read. Hillen A newly developed compound starves cancer cells by attacking their “power plants” – the so-called mitochondria.

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Gene Expression Genetics Gene Drugs 95

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

Scienmag

MAY 27, 2021

Cellular proteins get their marching orders from genetic instructions encoded in DNA, whose sequences are first copied and made […].

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Gene Expression RNA DNA Protein 41

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

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.

Gene Expression 52

Gene Expression Gene DNA RNA 52

Drug Discovery World

APRIL 17, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

Genome 52

Genome Genomics Gene Expression RNA 52

Scienmag

NOVEMBER 9, 2020

Preeclampsia-related gene expression in the placenta may correlate with vitamin D status during pregnancy, report researchers from the Medical University of South Carolina. Credit: Medical University of South Carolina According to the World Health Organization, preeclampsia affects between 2% to 8% of pregnancies.

Gene 52

Gene Gene Expression Research Cardiology 52

Drug Discovery World

OCTOBER 4, 2023

Scientists have discovered the link between gene expression, DNA methylation, and brain structural changes in depression, revealing new potential therapeutic targets. Parallel findings in humans underscored the involvement of interferon-related genes in major depressive disorder.

Research 52

Research Gene DNA Gene Expression 52

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

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. He added: “We have seen no significant gene misregulation attributable to the insertion event.

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Gene Therapy Gene Trials Gene Expression 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

Drug Discovery World

APRIL 3, 2024

Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Genome-wide screens are becoming the standard for drug discovery to power unbiased identification of drug targets across gene regulatory networks.

Genome 52

Genome Genomics Gene Expression RNA 52

pharmaphorum

OCTOBER 12, 2021

The research has focused on Alzheimer’s associated with an apolipoprotein E4 (APOE4) gene variant known to be a major risk factor for late-onset forms of the disease. The results are strong enough to back a proof-of-concept study in people with genetic risk of Alzheimer’s according to the researchers.

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Gene Expression Drugs FDA Approval Gene 111

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

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Genetics Medicine Gene Therapy Marketing 52

Scienmag

SEPTEMBER 29, 2020

By changing gene expression first, some immunotherapies may work better, models show Credit: (courtesy of Hackensack Meridian Health) September 29, 2020 – Nutley, NJ – A one-two punch of changing gene expression, then deploying immune checkpoint inhibitors, shows promise in battling one of the most treatment-resistant types of cancer in (..)

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Gene Expression Scientist Gene 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. The barcode sequence is followed by a unique molecular identifier, which enables accurate digital counting of gene transcripts.

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Gene Expression Development RNA Drugs 112

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

Drug Discovery World

OCTOBER 10, 2023

Read more: First oral drug for postpartum depression approved in US The role of genetics In October, scientists discovered the link between gene expression, DNA methylation, and brain structural changes in depression, revealing new potential therapeutic targets.

Drugs 59

Drugs Gene Expression Gene Immune Response 59

Scienmag

FEBRUARY 23, 2021

Credit: Sorbonne Université Using an innovative computational approach to analyze vast brain cell gene expression datasets, researchers at MIT and Sorbonne Université have found that Huntington’s disease may progress to advanced stages more because of a degradation of the cells’ health maintenance systems than because of increased damage (..)

Gene Expression 61

Gene Expression Gene Research Genetics 61

pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets. Dr Danuta Jeziorska.

Genome 59

Genome Genomics Gene Gene Expression 59

pharmaphorum

NOVEMBER 12, 2021

It could also be made more powerful by incorporating personal genetic information and gene expression profiles in tissues such as the lungs, they suggest.

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Doctors Doctor Drugs Gene Expression 134