Learn More
The relative ease, speed, and biological scope of clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated Protein9 (Cas9)-based reagents for genomic manipulations are revolutionizing virtually all areas of molecular biosciences, including functional genomics, genetics, applied biomedical research, and agricultural biotechnology.(More)
Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are devastating neurodegenerative disorders with clinical, genetic, and neuropathological overlap. Hexanucleotide (GGGGCC) repeat expansions in a noncoding region of C9ORF72 are the major genetic cause of FTD and ALS (c9FTD/ALS). The RNA structure of GGGGCC repeats renders these(More)
An expanded hexanucleotide repeat in C9orf72 causes amyotrophic lateral sclerosis and frontotemporal dementia (c9FTD/ALS). Therapeutics are being developed to target RNAs containing the expanded repeat sequence (GGGGCC); however, this approach is complicated by the presence of antisense strand transcription of expanded GGCCCC repeats. We found that(More)
C9orf72 mutations are the most common cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Dipeptide repeat proteins (DPRs) produced by unconventional translation of the C9orf72 repeat expansions cause neurodegeneration in cell culture and in animal models. We performed two unbiased screens in Saccharomyces cerevisiae and(More)
The increasing burden of the world population on agriculture requires the development of more robust crops. Dissecting the basic biology that underlies plant development and stress responses will inform the design of better crops. One powerful tool for studying plants at the molecular level is the RNA-programmed genome editing system composed of a clustered(More)
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are neurodegenerative diseases with overlapping genetic factors and pathology. On the cellular level, a majority of ALS and FTD cases are characterized by nuclear clearance and cytoplasmic aggregation of otherwise nuclear proteins, TAR DNA-binding protein 43 (TDP-43), or fused in sarcoma.(More)
1,1,3 Tricyano-2-amino-1-propene (Triap) is a small molecule that has neurotrophic properties similar to nerve growth factor (NGF). Studies have shown that NGF increases choline acetyltransferase (ChAT) activity, the enzyme responsible for the synthesis of acetylcholine, in several cell lines and in the CNS of adult animals. To investigate whether Triap can(More)
Please note that in the event of publication, it is mandatory that authors include all relevant methodological and statistical information in the manuscript. z Please specify the following information for each panel reporting quantitative data, and where each item is reported (section, e.g. Results, & paragraph number). z Each figure legend should ideally(More)
1,1,3 Tricyano-2-amino-1-propene (Triap) is a small molecular weight compound which increases the rate of nerve and tissue regeneration in several experimental systems. Early experiments with this compound showed that, like nerve growth factor (NGF), Triap induced neurite formation in chick spinal ganglia. To assess the similarity between NGF and Triap, we(More)
  • 1