A5027162703- Genetics and Neurodevelopmental Disorders
- Autism Spectrum Disorder Research
- Hippo pathway signaling and YAP/TAZ
- Congenital heart defects research
- Cancer-related gene regulation
- MicroRNA in disease regulation
- Genomics and Chromatin Dynamics
- Advanced biosensing and bioanalysis techniques
- Cardiomyopathy and Myosin Studies
- RNA Interference and Gene Delivery
- RNA Research and Splicing
Stanford University
2017-2020
Howard Hughes Medical Institute
2014-2017
Massachusetts Institute of Technology
2014
Allen Institute
2014
MicroRNAs (miRNAs) and siRNAs have enormous potential as cancer therapeutics, but their effective delivery to most solid tumors has been difficult. Here, we show that a new lung-targeting nanoparticle is capable of delivering miRNA mimics lung adenocarcinoma cells in vitro genetically engineered mouse model based on activation oncogenic Kirsten rat sarcoma viral oncogene homolog (Kras) loss p53 function. Therapeutic miR-34a, p53-regulated tumor suppressor miRNA, restored miR-34a levels...
Autism spectrum disorder (ASD) is a heterogeneous disease, but genetically defined models can provide an entry point to studying the molecular underpinnings of this disorder. We generated germline mutant mice with loss-of-function mutations in Chd8, de novo mutation strongly associated ASD, and demonstrate that these display hallmark ASD behaviors, macrocephaly, craniofacial abnormalities similar patient phenotypes. Chd8+/– broad, brain-region-specific dysregulation major regulatory cellular...
Significance The chromatin remodeler CHD8 is one of the most frequently mutated genes in autism spectrum disorder (ASD), but mechanistic basis remains unclear. Here, we identify dosage-sensitive roles for regulation transcription and define CHD8’s role regulating genome-wide accessibility. Importantly, present new results that help to molecular function both context pluripotency neural differentiation with implications its ASD. By determining execution point at which mutations Chd8 might...