Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …

Achieving sufficient delivery across the blood–brain barrier is a key challenge in the
development of drugs to treat central nervous system (CNS) disorders. This is particularly …

Despite the rising global incidence of central nervous system (CNS) disorders, CNS drug
development remains challenging, with high costs, long pathways to clinical use and high …

Loss-of-function heterozygous mutations in GRN, the gene encoding progranulin (PGRN),
were identified in patients with frontotemporal lobar degeneration (FTLD) almost two …

The blood-brain barrier (BBB) is one of the most essential protection mechanisms in the
central nervous system (CNS). It selectively allows individual molecules such as small lipid …

The blood brain barrier (BBB) presents a formidable challenge to the delivery of drugs into
the brain. Several strategies aim to overcome this obstacle and promote efficient and …

The vertebrate vasculature displays high organotypic specialization, with the structure and
function of blood vessels catering to the specific needs of each tissue. A unique feature of …

Effective delivery of protein therapeutics to the central nervous system (CNS) has been
greatly restricted by the blood-brain barrier (BBB). We describe the development of a BBB …

Alzheimer's disease (AD) is a multifactorial neurodegenerative disease which is mainly
characterized by progressive impairment in cognition, emotion, language and memory in …

The oligonucleotide therapeutics field has seen remarkable progress over the last few years
with the approval of the first antisense drug and with promising developments in late stage …