Drug Discovery World

SEPTEMBER 5, 2022

‘Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’. The goal of this final phase is to produce and phenotype hundreds of knockout mouse models for genes with little to no known function, to better understand the genetic bases for diseases in humans and animals. .

Gene Silencing 52

Gene Silencing Gene Genetics Genome 52

Drug Discovery World

OCTOBER 4, 2022

DDW Editor Reece Armstrong explores some of the trends that will be featured in sessions throughout ELRIG Drug Discovery 2022 and the news behind them. Automated systems have become a prominent feature throughout drug discovery, helping scientists save time and assisting in reproducible experiments for more verifiable results.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). After further discussion with Orchard Therapeutics, the NHS negotiated an additional, undisclosed discount on the drug, allowing the health agency to now offer it to patients.

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 103

In-Vivo Gene Editing Trials Drugs 103

Drug Discovery World

AUGUST 1, 2023

The NIH states: “This natural mechanism for sequence-specific gene silencing promises to revolutionise experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas.” There are many examples of ways in which RNAi is impacting drug discovery.

RNA 52

RNA Drugs Gene Silencing Vaccination 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. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage.

Gene Editing 52

Gene Editing Gene Engineer DNA 52