The nucleic acid therapeutics field has made tremendous progress in the past decades.
Continuous advances in chemistry and design have led to many successful clinical …

Nucleic acids drugs have been proven in the clinic as a powerful modality to treat inherited
and acquired diseases. However, key challenges including drug stability, renal clearance …

Antisense oligonucleotides are a relatively new therapeutic modality and safety evaluation is
still a develo** area of research. We have observed that some oligonucleotides can …

Rare diseases affect almost 500 million people globally, predominantly impacting children
and often leading to significantly impaired quality of life and high treatment costs. While …

Parkinson's disease (PD) is one of the most common neurodegenerative disorders that
manifest various motor and nonmotor symptoms. Although currently available therapies can …

Usher syndrome is a syndromic form of hereditary hearing impairment that includes
sensorineural hearing loss and delayed-onset retinitis pigmentosa (RP). Type 1 Usher …

In Huntington disease, cellular toxicity is particularly caused by toxic protein fragments
generated from the mutant huntingtin (HTT) protein. By modifying the HTT protein, we aim to …

Antisense oligonucleotides (ASOs) are promising therapeutics for intractable central
nervous system (CNS) diseases. For this clinical application, neurotoxicity is one of the …

Objectives Duchenne muscular dystrophy is associated with various degrees of cognitive
impairment and behavioral disturbances. Emotional and memory deficits also constitute …

Nucleic acid-based therapies have demonstrated great potential for the treatment of
monogenetic diseases, including neurologic disorders. To date, regulatory approval has …