The possibility of rational design and the resulting faster and more cost-efficient
development cycles of nucleic acid–based therapeutics (NBTs), such as antisense …

There are an estimated 7,000 “orphan diseases,” but treatments are currently available for
only about 5% of them. Recent progress in the advanced platforms of gene therapy, stem …

Orphan diseases are rare diseases that affect less than 200000 individuals within the United
States. Most orphan diseases are of neurologic and genetic origin. With the current …

Antisense oligonucleotide (ASO) therapies present a promising disease-modifying treatment
approach for rare neurological diseases (RNDs). However, the current focus is on “more …

The regulatory approvals of nusinersen and tofersen, plus the large body of clinical and
preclinical data from other drugs, have significantly de-risked antisense technology for …

The development of new sequencing technologies in the post-genomic era has accelerated
the identification of causative mutations of several single gene disorders. Advances in cell …

Antisense offers a precise and specific means of knocking down expression of a target gene,
and is a major focus of research in neuroscience and other areas. It has application as a tool …

The rising median age of our population and the age-dependent risk of neurodegeneration
translate to exponentially increasing numbers of afflicted individuals in the coming years …

Research into the genetic etiology of a neurological disorder can provide directions for
genetic diagnosis and targeted therapy. In the past, germline mutations, which are …

The advent of stem cell-derived cerebral organoids has already advanced our
understanding of disease mechanisms in neurological diseases. Despite this, many remain …