Contents
Vol 5, Issue 189
Contents
Research Articles
- In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous
Injection of a new gene therapy vector into the easily accessible vitreous transduced the entire retina and rescued disease phenotypes.
- Increased in Vivo Amyloid-β42 Production, Exchange, and Loss in Presenilin Mutation Carriers
Aβ42 kinetics are altered in the central nervous system of patients with autosomal dominant Alzheimer’s disease.
- Therapeutic Targeting of a Robust Non-Oncogene Addiction to PRKDC in ATM-Defective Tumors
Treating ATM-deficient cancers with an inhibitor of DNA-PKcs induces apoptosis due to inability to repair double-strand breaks in DNA.
State of the Art Review
- Genomic Medicine: A Decade of Successes, Challenges, and Opportunities
This State of the Art Review highlights the ways in which genomic tools and information are being used in clinical medicine, the big challenges in their uptake and implementation, and the forces that will shape genomic medicine in the future.
Editors' Choice
- Clearing the Air About Stroke
Short-term exposure to air pollutionis associated with lowered blood flow in the brain.
- A TWO Hit Wonder for Melanoma Treatment
A phase 1 clinical trial combining two immunotherapies improves response in advanced metastatic melanoma.
- Autism in the Balance
A gene linked to autism can drive both pathological and compensatory changes in the balance of excitatory and inhibitory neuronal activity.
- Making Sugar from Fat?
Fat cells cannot produce glucose, but they instruct the liver to do so by releasing a hormone called aP2.
Erratum
