The increasing number of approved nucleic acid therapeutics demonstrates the potential to
treat diseases by targeting their genetic blueprints in vivo. Conventional treatments …

Antisense technology is now beginning to deliver on its promise to treat diseases by
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …

Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Loss of nuclear TDP-43 is a hallmark of neurodegeneration in TDP-43 proteinopathies,
including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP-43 …

In the past decade, evidence for a fluid clearance pathway in the central nervous system
known as the glymphatic system has grown. According to the glymphatic system concept …

Recent medical advances have exploited the ability to address a given disease at the
underlying level of transcription and translation. These treatment paradigms utilize nucleic …

Timothy syndrome (TS) is a severe, multisystem disorder characterized by autism, epilepsy,
long-QT syndrome and other neuropsychiatric conditions. TS type 1 (TS1) is caused by a …

Huntington disease (HD) is a neurodegenerative disease caused by CAG repeat expansion
in the huntingtin gene (HTT) and involves a complex web of pathogenic mechanisms …

With the 2019 breakthrough in the development of highly effective modulator therapy
providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who …

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder caused by the loss
of motor neurons from the brain and spinal cord. The ALS community has made remarkable …