Contents
21 August 2019
Vol 11, Issue 506
Vol 11, Issue 506
Focus
- Neoantigen quality, not quantity
Prioritizing expressed clonal neoantigens in genes required for cancer cell survival may reduce the likelihood of resistance to neoantigen therapies.
Research Articles
- Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A
Gene therapy for Niemann-Pick type A disease is safe in nonhuman primates and has therapeutic effects in a mouse model.
- The autophagy-activating kinase ULK1 mediates clearance of free α-globin in β-thalassemia
Rapamycin alleviates β-thalassemia by stimulating ULK1-dependent clearance of toxic free α-globin.
- Imaging-assisted nanoimmunotherapy for atherosclerosis in multiple species
A nanoimmunotherapy reduces inflammation in mouse, rabbit, and pig models of atherosclerosis.
- Treating murine inflammatory diseases with an anti-erythrocyte antibody
An anti-erythrocyte antibody redirects inflammation in multiple mouse models.
Editors' Choice
- Alternative rhythms in schizophrenia
A distinct set of rhythmically expressed genes related to mitochondrial function is identified in the brains of patients with schizophrenia.
- A face-lift for reconstructive surgery
3D printing and digital modeling improve face transplantation for people affected by facial trauma unrepairable by traditional reconstructive surgery.
- Blaming an unusual suspect for endosomal traffic jams in Alzheimer’s disease
Accumulation of β–C-terminal fragments, not β-amyloid, drives early endosomal alterations in familial Alzheimer’s disease human neurons.
- Interrogating CD8+ T cell reactivity on a genome-wide scale
A new method enables large-scale identification of human T cell antigens.
Erratum
About The Cover
ONLINE COVER Widespread Benefits. Patients with Niemann-Pick disease type A (NPD-A) develop multi-organ pathologies. Within the brain, NPD-A triggers neurodegeneration in several regions, including the cerebellum (depicted here). NPD-A is caused by loss-of-function mutations in the gene encoding acid sphingomyelinase (ASM). Because prior research has shown that intracerebral gene therapy using adeno-associated viral vector (AAV) is not distributed beyond the area surrounding the injection site, Samaranch et al. delivered AAV-ASM through the cerebellomedullary cistern (CM). CM injection of AAV9-ASM in nonhuman primates resulted in widespread gene distribution throughout the nervous system, without signs of toxicity. In a mouse model of NPD-A, treatment ameliorated liver pathology and reduced neurodegeneration in several brain areas. [CREDIT: SAMARANCH ET AL./SCIENCE TRANSLATIONAL MEDICINE]
