The Pharma Data

MARCH 3, 2022

In 2014, Shalev’s UAB research lab reported that verapamil completely reversed diabetes in animal models, and she announced plans to test the effects of the drug in a human clinical trial. Fifty-three proteins showed significantly altered relative abundance over time in response to verapamil.

Insulin 52

Insulin Gene Expression Gene Protein 52

Drug Discovery World

DECEMBER 12, 2022

In Nature 3 , we learnt about the CRISPR cancer trial success leading the way to personalised treatments. 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.

Drugs 52

Drugs Gene Editing Genome Genomics 52

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. Most of them are rare mutations.”.

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Gene Editing Gene Gene Therapy In-Vivo 144

Drug Discovery World

APRIL 30, 2024

Its job is to carry coding information that is essential to the translation and processing of functional proteins. This is essential to its use as a therapeutic agent and gives the technology a vast versatility making it suitable to treating a wide range of diseases – especially those that have high protein expression.

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RNA Protein Vaccination Vaccine 52

Drug Discovery World

MARCH 26, 2024

UCDs are a group of rare, severe, inherited metabolic diseases impacting protein metabolism. Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 targets carbamoyl phosphate synthetase 1 (CPS1), a key enzyme that catalyses the first step of the urea cycle.

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Trials Gene Expression Protein Gene 52

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

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

Drug Discovery World

OCTOBER 17, 2023

Today, spatial omics technologies enable imaging-based identification of cells in their native tissue context while simultaneously detecting hundreds of RNAs or tens of protein markers in the same cells. There is a lack of data and case studies beyond pilot projects related to patient recruitment and clinical trials.

Gene Expression 52

Gene Expression Drugs Protein Gene 52

Drug Discovery World

FEBRUARY 6, 2023

Typically, the data obtained from such studies is large and high dimensional with thousands of molecules being measured for each individual sample requiring special statistical, data science, and bioinformatics skills to integrate the information and correlate it with the clinical outcome of interest.

Big Data 52

Big Data RNA Bioinformatics Vaccination 52

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

Marketing 52

Marketing Gene Drugs Protein 52

XTalks

MARCH 7, 2024

Experts discuss the therapeutic landscape, treatment gaps, regulatory considerations and clinical trial strategies. This on-demand webinar explores the perinatal period and related psychiatric conditions, focusing on perinatal mood disorders. Through this, researchers can pinpoint breast cancer cell types and their phenotypic states.

Hormones 115

Hormones Genetics Genome Genomics 115

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

The Pharma Data

DECEMBER 27, 2020

Even with the holidays among us, there were a number of clinical trial announcements. Vir Biotechnology and GlaxoSmithKline dosed the first patient in a new sub-trial of a Phase III study of monoclonal antibody VIR-7831 for hospitalized adults with COVID-19. Here’s a look. COVID-19-Related.

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Trials Antibody Gene Therapy Clinical Trials 52

Drug Discovery World

JANUARY 23, 2023

Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2. The trial saw positive results from grafting a sheet of retinal pigment epithelial cells differentiated from iPSCs into the eye of a patient with macular degeneration 8.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

DECEMBER 15, 2022

Now, after nearly 35 years of research and more than a decade of preclinical progress, several different gene editing modalities are being tested in early phase clinical trials. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

XTalks

DECEMBER 24, 2020

SARS-CoV-2 is characterized by an outer surface speckled with spike proteins, giving it a crown-like appearance, hence the name “corona,” which is the Latin word for crown. The spike protein is critical for binding to ACE2 receptors on host cells — this interaction mediates the entry of the virus into cells. CRISPR Therapeutics.

Life Science 111

Life Science Vaccination Vaccine Gene 111

The Pharma Data

AUGUST 3, 2021

HDACs catalyze the removal of acetyl groups from acetylated lysine residues in histones, resulting in the modulation of gene expression. HDACs also deacetylate non-histone proteins, such as transcription factors. Reactivation of hepatitis B virus infection was reported in 1% of patients in clinical trials.

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Nurses In-Vitro Gene Expression Medicine 52

The Pharma Data

MAY 13, 2021

based weight loss intervention in adults with obesity: A randomized clinical trial. Hepatic ALT isoenzymes are elevated in gluconeogenic conditions including diabetes and suppressed by insulin at the protein level. Opiate-like effects of sugar on gene expression in reward areas of the rat brain. Journal of Obesity.

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Insulin Production Hormones Medicine 52