pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 86

Genome Genomics Genetics Genome Project 86

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 73

Genome Genomics Genetics Genome Project 73

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genome 98

Genome Genomics Medicine Genetic Disease 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 through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

AUGUST 29, 2022

Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.

Gene Editing 98

Gene Editing DNA Gene 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

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

Drug Discovery World

OCTOBER 12, 2023

New approaches and thought processes When it comes to diversity in drug discovery, this includes our scientists’ and pharma workforce’s approach to innovation which will help advance the industry. Dr Michelle Fraser is Head of Cell & Gene Therapy at global health science solutions company, Revvity.

Drugs 59

Drugs Life Science Genome Genomics 59

Drug Discovery World

APRIL 30, 2024

mRNA exists in every cell within the human body and this, along with its role in the development of proteins, are the reasons why mRNA therapies have such potential in treating many diseases. Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells.

RNA 52

RNA Protein Vaccination Vaccine 52