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Researchers reveal how microbiome bacteria adapt through transmission
Scientists have used genetic sequencing to demonstrate how the transmission of gut bacteria influences its evolution and functions, which could effect human health.
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Genomics
Genomics is the branch of molecular biology concerned with the structure, function, evolution, and mapping of genomes.
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Therapeutic target for aggressive cancers discovered through CRISPR
The RNA-modifying protein METTL1 could be targeted to treat some types of aggressive cancers, including brain, blood and kidney.
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CRISPR-Cas9 used to prevent Fuchs’ corneal dystrophy in mice
Scientists have shown that start codon disruption with CRISPR-Cas9 gene editing can prevent Fuchs’ corneal dystrophy in mouse models.
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Genes behind antibiotic activity in bacteria uncovered by metabologenomics
Researchers have used comparative metabologenomics to uncover what may be “silencing” bacteria to produce desirable compounds.
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Removal of RNA-binding protein improves leukaemia survival
Study shows that removing the protein IGF2BP3 slows cancer growth and increases chances of survival of rare types of leukaemia in mice.
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Novel group of small molecules show promise against SARS-CoV-2
Researchers in Germany have identified 69 small molecules as binding partners for genomic RNA of SARS-CoV-2, possibly leading to new drugs.
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Prenatal editing halts progression of Hurler syndrome in pre-clinical model
US researchers used an AAV9 vector to edit a single base mutation in a prenatal mouse model, halting progression of Hurler syndrome.
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Defective proteins found to be potential targets for Alzheimer’s treatments
US researchers have identified potential new treatment targets for Alzheimer’s disease, as well as existing drugs that could be used against these targets.
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Exome sequencing reveals genetic drug target for OCD
Following a whole exome sequencing study, researchers have found that mutations in the SLITRK5 gene could be targeted by drugs to treat OCD.
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Novel vectors for more efficient and less risky ocular gene therapy
Inherited blindness is the leading cause of vision loss in the working populations. Recently, the first gene therapy for the treatment of retinal dystrophy caused by mutations in the RPE65 gene became available. This gene therapy is based on adeno-associated virus (AAV) vectors injected under the retina. Subretinal injection is…
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The potential of epigenomic medicine in cardiology
Advances in genomic medicine are playing an increasingly important role in the field of cardiology. Better analysis and understanding of patient genomic and epigenomic information can enable more personalised patient treatment and medical intervention. Here, Professor John Giannios considers the potential to use genomic medicine to prevent, monitor, diagnose and…
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Reference genome comparison finds exome variant discrepancies
Scientists have found differences in 206 genes between the GRCh38 (hg38) and GRCh37 (hg19) human reference genomes.
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Drug Target Review – Issue 2 2021
This issue includes articles on novel AAV vectors to deliver ocular gene therapy, how phenotypic models of disease are being used in covalent fragment screening and the challenges and opportunities presented by automation in the life sciences. Also in this issue are features on stem cells, antibodies and hit-to-lead.
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Challenges and opportunities in gene therapy development
Despite the promise of gene therapies, significant challenges have emerged in the field. Dr Carsten Brunn discusses the current obstacles and opportunities when developing gene therapies.
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SARS-CoV-2 “Mexican variant” sequence analysed by researchers
Analysis of one million SARS-CoV-2 genome sequences has revealed a new variant named T478K, spreading mostly in Mexico.
