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Host-cell factors involved in COVID-19 infections may predict improved treatments
Researchers have found a potential therapeutic target less vulnerable to potential drug resistance and emerging COVID-19 variants of concern.
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New system for safer CRISPR-based gene drive testing and development
US researchers outline the development of a new system for testing and developing CRISPR-based gene drives in the laboratory, and safely converting them into tools for potential real-world applications.
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New study highlights lipid nanoparticles and mRNA could treat blindness
The researchers developed nanoparticles able to penetrate the neural retina and deliver mRNA to the photoreceptor cells whose proper function makes vision possible.
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Study identifies gene and proteins that could treat dry eye disease
Researchers found that proteins made by stem cells that regenerate the cornea could be new targets for treating dry eye disease.
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Novel protein coordinates melanin production
Japanese researchers reported that a key transcription factor “Nrf3” regulates the process of melanin production in mouse and human cells.
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Five recent cell and gene therapy discoveries
This article highlights five of the latest findings using cell and gene therapy techniques that could be used in the development or design of new therapies.
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Scientists investigate the multi-omic landscape of NAFLD-to-NASH progression
Researchers have presented comprehensive multi-omic profiles to identify genes, non-coding RNAs, proteins, and plasma metabolites involved in NAFLD-to-NASH progression.
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Study in mice shows dietary saturated fats can trigger liver cancer
Japanese researchers showed for the first time processes that are crucial targets for treating liver cancer are enhanced by saturated fatty acid diets.
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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).
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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…
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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).
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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.
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Pattern of gene activity for ADHD is unlocked
US researchers identified that those with ADHD have differences in gene activity in the brain.
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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.
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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.
