Contents
11 November 2015
Vol 7, Issue 313
Vol 7, Issue 313
Contents
Editorial
- Hearing voices: FDA seeks advice from patients
A new patient-engagement committee will advise the U.S. Food and Drug Administration on the regulation and use of medical devices.
Focus
- Regulatory decision-making meets the real world
As patient input in drug development increases and new data sources are tapped, regulators need to organize and ensure the quality of data to inform decision-making.
Research Articles
- A validated gene regulatory network and GWAS identifies early regulators of T cell–associated diseases
Combining a gene regulatory network and disease association data identified early regulators of T cell–associated diseases.
- Virtual typing by people with tetraplegia using a self-calibrating intracortical brain-computer interface
Individuals with tetraplegia are able to type self-paced for hours across multiple days using a self-calibrating point-and-click intracortical brain-computer interface.
- AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease
AAV-mediated gene transfer to ependymal cells in a dog model of Batten disease provides sustained enzyme replacement and delays disease onset.
- Modeling pulmonary alveolar microlithiasis by epithelial deletion of the Npt2b sodium phosphate cotransporter reveals putative biomarkers and strategies for treatment
Epithelial deletion of Npt2b results in a tractable mimic of pulmonary alveolar microlithiasis.
- Analysis of ESR1 mutation in circulating tumor DNA demonstrates evolution during therapy for metastatic breast cancer
ESR1 mutations evolve during the treatment of metastatic breast cancer.
Editors' Choice
- Wound healing goes green
Biomaterials containing photosynthetic algae promote tissue regeneration in a mouse model.
- Singled out: Exploring epigenetics
Droplet-based microfluidics and DNA barcoding reveal the epigenetic state of chromatin in single cells.
About The Cover
ONLINE COVER Gene Therapy Lights Up. Shown is expression of recombinant enzyme (pseudo-colored blue) in the hippocampus of a dog with Batten disease after a single gene therapy treatment. In this naturally occurring dog model of a lysosomal storage disease, deficiency of the lysosomal enzyme TPP1 results in motor abnormalities, cognitive deficits, seizures, and vision loss. One-time delivery of the gene encoding the canine form of TPP1 (via an adeno-associated viral vector) to the cerebral ventricles of this dog model resulted in delayed disease progression, protection from cognitive decline and extended life span. (Katz et al.). [CREDIT: L. TECEDOR AND B. DAVIDSON/THE CHILDREN'S HOSPITAL OF PHILADELPHIA]