Drug Discovery World

APRIL 17, 2024

JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. Recently, research published by Dr Richard (Rick) Young (Whitehead Institute) describes the key mechanism of how these regRNAs regulate gene expression. What diseases are you currently targeting?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

pharmaphorum

SEPTEMBER 6, 2021

The biotech said that the US regulator ordered the pause on the study after liver tumours were seen in mice given the therapy in preclinical testing. PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine, an amino acid that is commonly found in many foods.

Gene Therapy 84

Gene Therapy Gene Genetic Disease DNA 84

The Pharma Data

OCTOBER 18, 2020

(NASDAQ:CDXC) today highlighted a new study published in The European Molecular Biology Organization Journal looking at the effect of nicotinamide riboside (NR) on maintaining telomeres, the protective regions at the end of DNA strands. Telomeres are “caps” at the end of chromosomes that protect DNA from getting worn away as cells replicate.

DNA 52

DNA Scientist Genetic Disease Research 52

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.

RNA 52

RNA Protein Vaccination Vaccine 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 through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

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

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64