Pharmaceutical Technology

FEBRUARY 20, 2023

While there is no cure to this neurodegenerative condition, academics and companies are pushing through with research that could help patients and their families. Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials.

Development 246

Development Gene Therapy Protein Gene 246

Scienmag

JANUARY 4, 2021

BOSTON – A long-running debate over how an important gene-silencing protein identifies its targets has been resolved by researchers at Massachusetts General Hospital (MGH). The findings could yield important implications for development of drugs to treat cancer and other diseases.

Gene Silencing 41

Gene Silencing Gene Protein Development 41

Scienmag

JULY 2, 2021

The ongoing effort to explore […].

Gene Silencing 75

Gene Silencing Gene Editing Gene DNA 75

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

Scienmag

JUNE 7, 2021

Divita Mathur, Research Assistant Professor, is studying cytosolic access and instability of DNA nanoparticles. A number of candidate therapies such as CRISPR-Cas9 and gene silencing require the efficient delivery of functional nucleic acids to the cell cytosol and nucleus.

DNA 51

DNA Gene Silencing Gene Research 51

pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 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. It’s a great tool for controlling gene expression.”.

DNA 98

DNA Gene Gene Silencing Genome 98