Repetitive elements in the human genome, once considered 'junk DNA', are now known to
adopt more than a dozen alternative (that is, non-B) DNA structures, such as self-annealed …

Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …

Huntington's disease (HD) is characterized by preferential loss of the medium spiny neurons
in the striatum. Using CRISPR/Cas9 and somatic nuclear transfer technology, we …

Approximately 70 human RNA-binding proteins (RBPs) contain a prion-like domain (PrLD).
PrLDs are low-complexity domains that possess a similar amino acid composition to prion …

Although it was long thought that eukaryotic translation almost always initiates at an AUG
start codon, recent advancements in ribosome footprint map** have revealed that non …

The monogenic nature of Huntington's disease (HD) and other neurodegenerative diseases
caused by the expansion of glutamine-encoding CAG repeats makes them particularly …

Expression of many disease-related aggregation-prone proteins results in cytotoxicity and
the formation of large intracellular inclusion bodies. To gain insight into the role of inclusions …

Neurodegenerative and neuronal-related diseases are major public health concerns.
Human vulnerability to neurodegenerative diseases (NDDs) increases with age. Neuronal …

To define how the C9orf72 GGGGCC expansion mutation causes ALS/FTD and to facilitate
therapy development, a mouse model that recapitulates the molecular and phenotypic …

Biomolecules are in constant motion. To understand how they function, and why
malfunctions can cause disease, it is necessary to describe their three-dimensional …