news
Novel gene therapy offers potential treatment for FCMD
Japanese researchers proposed using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama Muscular Dystrophy (FCMD).
List view / Grid view
RNAs
article
Silencing the messenger in elevated lipoprotein(a)
Elevated lipoprotein(a), or Lp(a), is a major risk factor for cardiovascular disease that affects one in five people worldwide, but currently lacks approved therapies. Here, Dr Giles Campion, EVP, Head of R&D and Chief Medical Officer of Silence Therapeutics, describes the company’s approach to developing an investigational siRNA therapy designed…
article
Drug Target Review Cell and Gene Therapy Supplement 2022
This supplement focuses on how cell and gene therapy can target diseases such as cancer and reduce elevated lipoprotein(a).
news
Genetic cause of ALS and dementia repaired by RNA-targeting strategy
US scientists have developed a potential medication for the genetic cause of ALS and dementia, that eliminates the mutated segments of RNA.
news
Pattern of gene activity for ADHD is unlocked
US researchers identified that those with ADHD have differences in gene activity in the brain.
article
How ageing triggers Huntington’s disease
A new study found that as patients age, Huntington’s disease gradually impairs the important cellular housekeeping process autophagy, which is responsible for eliminating waste from cells.
news
New treatment target discovered for sleep apnoea
A recent study in mice revealed that an ion channel already proven to affect blood pressure in obese mice plays a role in suppressing their breathing, influencing sleep apnoea.
news
RNA: the key to cancer research?
In the production of RNA, researchers in Spain and Denmark have found a method for attacking cancer cells.
news
Lipid nanoparticle delivery of mRNA could lead to new ‘universal’ COVID-19 treatment
A study has shown that mRNA delivered via lipid nanoparticles blocks multiple variants of SARS-CoV-2 from entering cells in mice.
article
Is an HIV vaccine on the horizon?
Researchers in the US have developed a potential HIV vaccine approach that aims to prompt the creation of broadly neutralising antibodies via mRNA.
news
New study provides insight for how congenital heart defects manifest
The researchers have found that the cells lining the heart direct the cardiac muscle to grow until the heart reaches its full size.
news
Study reveals potential therapeutic targets for pancreatic cancer
Researchers analysed the expression of long non-coding RNAs in samples from patients and tumour cell lines, identifying a group of genes with augmented expression in pancreatic cancer.
news
Screening technique could accelerate and improve mRNA therapies
A new single-cell nanoparticle targeting-sequencing approach can measure the cellular uptake of lipid nanoparticles carrying mRNA therapies.
news
Broadly neutralising antibody protects Syrian hamsters against Omicron variants
Researchers have found that a broadly neutralising antibody displays strong antiviral activities against Omicron BA.1, BA1.1 and BA.2.
article
ELRIG: New modalities in pharmacology and drug discovery
Drug Target Review’s Ria Kakkad recently visited ELRIG and the British Pharmacological Society’s conference on New Modalities in Pharmacology and Drug Discovery which took place in London, UK. This article shares some of the key takeaways from the event.
