Contents
15 May 2019
Vol 11, Issue 492
Vol 11, Issue 492
Review
- Advances in therapeutic applications of extracellular vesicles
Understanding extracellular vesicle biology and production will pave the way for a new type of biotherapeutic.
Research Articles
- Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina
Adeno-associated viral vectors reconstitute large proteins in the retina via intein-mediated protein trans-splicing.
- Detecting middle ear fluid using smartphones
To diagnose otitis media, a smartphone system detects middle ear fluid using the built-in microphone and speakers.
- A virus-like particle vaccine prevents equine encephalitis virus infection in nonhuman primates
A multivalent VLP vaccine protects against aerosol challenge of three lethal encephalitis viruses in mice and nonhuman primates.
- Human sebum requires de novo lipogenesis, which is increased in acne vulgaris and suppressed by acetyl-CoA carboxylase inhibition
De novo lipogenesis is essential for human sebum production, and increased lipogenic flux accounts for excessive sebum production in acne vulgaris.
- Cediranib suppresses homology-directed DNA repair through down-regulation of BRCA1/2 and RAD51
Cediranib directly suppresses homology-directed DNA repair in tumor cells and confers synthetic lethal vulnerability to PARP inhibitors.
Editors' Choice
- Organs-on-chips take baby steps
Two organ-on-chip devices enable investigation of cellular interactions in human fetal membranes in vitro.
- SHANK3 puts autism to sleep
Mutation of autism-associated gene SHANK3 leads to sleep problems in mice and humans.
- CuRBing cancer’s enthusiasm
A gene expression signature of biallelic RB1 loss identifies more aggressive tumors.
- Mind the gap: Cx32 and α-synuclein
α-synuclein oligomer uptake by neurons and oligodendrocytes exploits connexin-32.
About The Cover
ONLINE COVER Going Big in Gene Therapy. The image shows a three-dimensional human retinal organoid co-transduced by two adeno-associated viral (AAV) vectors. Tornabene et al. tested the efficiency of producing large proteins using AAV split intein-mediated protein trans-splicing to treat inherited retinal diseases. Inteins are protein segments that self-excise and rearrange spontaneously into large proteins. The AAV vectors were safe and improved the phenotypes of mouse models of Stargardt disease and Leber congenital amaurosis type 10. These findings support the therapeutic potential of AAV intein vectors for gene therapy of blinding diseases due to mutations in large genes. [CREDIT: TORNABENE ET AL./SCIENCE TRANSLATIONAL MEDICINE]