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.

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Scienmag

JUNE 14, 2021

The study, “Transcriptomics identifies STAT3 as a key regulator of hippocampal gene expression and anhedonia during withdrawal from chronic alcohol exposure,” is published in the journal Translational Psychiatry by researchers […].

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Scienmag

JANUARY 11, 2021

These biomarkers are epigenetic, meaning they involve changes to molecular factors that regulate genome activity such as gene expression independent of DNA sequence, and can be passed down to future generations. In a […].

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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.

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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. Shares in bluebird ticked up following the announcement.

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The Pharma Data

JULY 11, 2021

On the one hand, they can boost the metabolism of T cells by inducing central regulators of energy metabolism. On the other hand, they can inhibit specific enzymes that regulate the accessibility to the genetic material and thus the gene expression in the T cells. In doing so, they induce epigenetic changes.

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pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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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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Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. They are different from small molecules in terms of their size and complexity.

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Roots Analysis

AUGUST 31, 2023

It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

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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. Thus, targeting menin may be an effective strategy for patients with high unmet needs.

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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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Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Patients with argininosuccinic aciduria can also experience an imbalance of glutathione regulation, which is important for liver detoxification. We believe it can now do the same for rare diseases.” “We

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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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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.

DNA 52

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

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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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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Drug Discovery World

JULY 15, 2022

This three dimensional packing of DNA is crucial for gene regulation and for the DNA to fit into the cells because the total length of cellular DNA in cells is up to a hundred thousand times the cell’s length. iv) Highly repetitive regions contain some of the highest mutation rates.

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The Pharma Data

NOVEMBER 9, 2020

This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. Furthermore, Amgen’s research, testing, pricing, marketing and other operations are subject to extensive regulation by domestic and foreign government regulatory authorities.

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XTalks

DECEMBER 24, 2020

However, for virologists like Lori Frappier, PhD, professor in the department of molecular genetics at the University of Toronto, the outbreak of SARS-CoV-2 was less of a surprise and more of an inevitability. CRISPR Gene Editing Inventors Win Nobel Prize. Vaccine Considerations. But nevertheless, 2021 into 2022 will be the time.”.

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The Pharma Data

MAY 13, 2021

The trans-fat ban–food regulation and long-term health. Regulation of glucose metabolism from a liver-centric perspective. Neural and Genetic Correlates of the Social Sharing of Happiness. Adipose tissue regulates insulin sensitivity: role of adipogenesis, de novo lipogenesis and novel lipids. Biochem Cell Biol.

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The Pharma Data

MAY 15, 2021

All too often, it is lifestyle factors – the things we do day to day – that determines whether a particular gene switches on to create actual illness. The scientific study of how to undo genetic illnesses – like HCT – is called epigenetics. It’s because certain genes act one way on some people… and another way on other people.

Bacteria 52

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