Researchers at Oregon Health and Science University have used mouse models to estimate the frequency at which gene therapies delivered by adeno-associated virus (AAV) vectors integrated into host DNA, and come up with an estimate of up to roughly 3% – a number that is orders of magnitude higher than previous estimates and would translate into several hundred million cells with integrated viral vectors in an adult liver, assuming that 10% of cells took up the transgene.
In studies that give new insights into both developmental biology and the origins of melanoma, investigators at Memorial Sloan-Kettering Cancer Center and Weill Cornell Medical College have identified the activity of chromatin remodeling protein ATAD2 as necessary for cells with the oncogenic mutation V600E to give rise to melanomas. Involvement of epigenetic factors in cancers, or their targeting, is not new in cancer – as HDAC inhibitors as well as newer drugs such as the EZH2 inhibitor Tazverik (tazemetostat, Epizyme Inc.) demonstrate. But to Richard White and his colleagues, the point of their work is not so much about individual targets.
In studies that give new insights into both developmental biology and the origins of melanoma, investigators at Memorial Sloan-Kettering Cancer Center and Weill Cornell Medical College have identified the activity of chromatin remodeling protein ATAD2 as necessary for cells with the oncogenic mutation V600E to give rise to melanomas.
Investigators at MIT have identified a protein capable of delivering its own mRNA to cells, and engineered that protein to deliver mRNA sequences of their choosing. In a mouse model, the team used their approach to deliver the mRNA for two different proteins.
Researchers from Denali Therapeutics Inc. have identified new functional links between progranulin, lysosomal function, and a subtype of frontotemporal dementia caused by progranulin deficiency (FTD-GRN) that suggest progranulin-mediated FTD could be conceptualized as a lysosomal storage disorder (LSD). They also showed that delivery of their experimental therapeutic PTV:PGRN, also known as DNL-593, reduced cell damage and symptoms of FTD in cell and animal models.
A team of researchers has created peptide-like molecules – "peptoids" – with antiviral properties that could circumvent the naturally occurring antimicrobial peptides' shortcomings.
For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.
Investigators at MIT have identified a protein capable of delivering its own mRNA to cells, and engineered that protein to deliver mRNA sequences of their choosing.
For most people, neither polyglutamine disorders nor neuromuscular disorders are likely to be among the things they associate with androgen receptor (AR) dysfunction. But the three are indeed linked. And researchers have reported new insights into the nature of those links that could lead to a treatment for spinal and bulbar muscular atrophy, and possibly other disorders linked to AR signaling dysfunction.
