Parkinson's disease genetics and pathophysiology
Parkinson's disease (PD) is a common neurodegenerative disorder characterized by
degeneration of the substantia nigra pars compacta and by accumulation of α-synuclein in …
degeneration of the substantia nigra pars compacta and by accumulation of α-synuclein in …
Alpha-synuclein aggregation pathway in Parkinson's disease: current status and novel therapeutic approaches
Following Alzheimer's, Parkinson's disease (PD) is the second-most common
neurodegenerative disorder, sharing an unclear pathophysiology, a multifactorial profile …
neurodegenerative disorder, sharing an unclear pathophysiology, a multifactorial profile …
Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier
SJ Barker, MB Thayer, C Kim, D Tatarakis… - Science Translational …, 2024 - science.org
Antisense oligonucleotides (ASOs) are promising therapeutics for treating various
neurological disorders. However, ASOs are unable to readily cross the mammalian blood …
neurological disorders. However, ASOs are unable to readily cross the mammalian blood …
A single-cell map of antisense oligonucleotide activity in the brain
Antisense oligonucleotides (ASOs) dosed into cerebrospinal fluid (CSF) distribute broadly
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …
Therapeutics in the pipeline targeting α-synuclein for Parkinson's disease
Parkinson's disease (PD) is the second most common neurodegenerative disorder and the
fastest growing neurologic disease in the world, yet no disease-modifying therapy is …
fastest growing neurologic disease in the world, yet no disease-modifying therapy is …
Evaluating drug targets through human loss-of-function genetic variation
Naturally occurring human genetic variants that are predicted to inactivate protein-coding
genes provide an in vivo model of human gene inactivation that complements knockout …
genes provide an in vivo model of human gene inactivation that complements knockout …
Antisense drugs make sense for neurological diseases
CF Bennett, HB Kordasiewicz… - Annual review of …, 2021 - annualreviews.org
The genetic basis for most inherited neurodegenerative diseases has been identified, yet
there are limited disease-modifying therapies for these patients. A new class of drugs …
there are limited disease-modifying therapies for these patients. A new class of drugs …
Emerging therapeutic strategies for Parkinson's disease and future prospects: A 2021 update
Parkinson's disease (PD) is a neurodegenerative disorder pathologically distinguished by
degeneration of dopaminergic neurons in the substantia nigra pars compacta. Muscle …
degeneration of dopaminergic neurons in the substantia nigra pars compacta. Muscle …
The clinical drug candidate anle138b binds in a cavity of lipidic α-synuclein fibrils
Aggregation of amyloidogenic proteins is a characteristic of multiple neurodegenerative
diseases. Atomic resolution of small molecule binding to such pathological protein …
diseases. Atomic resolution of small molecule binding to such pathological protein …
Who ever said it would be easy? Reflecting on two clinical trials targeting α‐synuclein
Two recent, high‐profile manuscripts reported negative results with two parallel approaches
of passive immunization targeting α‐synuclein in a population of patients with early …
of passive immunization targeting α‐synuclein in a population of patients with early …