The present disclosure features useful compositions and methods to treat trinucleotide repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with OGG1 activity.

The present disclosure features useful compositions and methods to treat trinucleotide repeat expansion disorders, e.g., in a subject in need thereof. In some aspects, the compositions and methods described herein are useful in the treatment of disorders associated with OGG1 activity.

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.

The present invention relates to a double-stranded oligonucleotide capable of inhibiting CTGF expression with a very specific and high efficiency, a double-stranded oligonucleotide structure and nanoparticles comprising the double-stranded oligonucleotide, and a use thereof in preventing or treating of fibrotic or respiratory diseases.

The present invention relates to a double-stranded oligonucleotide capable of inhibiting CTGF expression with a very specific and high efficiency, a double-stranded oligonucleotide structure and nanoparticles comprising the double-stranded oligonucleotide, and a use thereof in preventing or treating of fibrotic or respiratory diseases.

The present disclosure relates to agents, compositions targeting to von Willebrand factor (VWF). The VWF targeting agents are synthetic polynucleotides, including VWF binding agents and their reversal agents. The VWF binding agents are VWF binding aptamers that bind to and inhibit the VWF activities. The VWF binding agents can be reversed using reversal agents to reverse the inhibitory effect and thereby restore VWF activities. The disclosure further provides methods for regulating the activities of VWF, thereby modulating VWF mediated platelet functionality, such as thrombosis. the present VWF targeting agents may be used for preventing thrombus formation and treating thrombotic disorders.

The present disclosure relates to agents, compositions targeting to von Willebrand factor (VWF). The VWF targeting agents are synthetic polynucleotides, including VWF binding agents and their reversal agents. The VWF binding agents are VWF binding aptamers that bind to and inhibit the VWF activities. The VWF binding agents can be reversed using reversal agents to reverse the inhibitory effect and thereby restore VWF activities. The disclosure further provides methods for regulating the activities of VWF, thereby modulating VWF mediated platelet functionality, such as thrombosis. the present VWF targeting agents may be used for preventing thrombus formation and treating thrombotic disorders.

Provided are compounds, methods, and pharmaceutical compositions for modulating SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral and developmental delays, movement and balance dysfunctions, orthopedic conditions, motor and cognitive dysfunctions, delayed language and speech issues, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, growth and nutrition issues, sleeping difficulties, chronic infections, sensory integration disorders, and dysautonomia.

Provided are compounds, methods, and pharmaceutical compositions for modulating SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral and developmental delays, movement and balance dysfunctions, orthopedic conditions, motor and cognitive dysfunctions, delayed language and speech issues, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, growth and nutrition issues, sleeping difficulties, chronic infections, sensory integration disorders, and dysautonomia.