دورية أكاديمية

Alpha-Synuclein Targeting Therapeutics for Parkinson's Disease and Related Synucleinopathies

التفاصيل البيبلوغرافية
العنوان: Alpha-Synuclein Targeting Therapeutics for Parkinson's Disease and Related Synucleinopathies
المؤلفون: Menon, Sindhu, Armstrong, Sabrina, Hamzeh, Amir, Visanji, Naomi P., Sardi, Sergio Pablo, Tandon, Anurag
المصدر: Frontiers in Neurology ; volume 13 ; ISSN 1664-2295
بيانات النشر: Frontiers Media SA
سنة النشر: 2022
المجموعة: Frontiers (Publisher - via CrossRef)
الوصف: α-Synuclein (asyn) is a key pathogenetic factor in a group of neurodegenerative diseases generically known as synucleinopathies, including Parkinson's disease (PD), dementia with Lewy bodies (DLB) and multiple system atrophy (MSA). Although the initial triggers of pathology and progression are unclear, multiple lines of evidence support therapeutic targeting of asyn in order to limit its prion-like misfolding. Here, we review recent pre-clinical and clinical work that offers promising treatment strategies to sequester, degrade, or silence asyn expression as a means to reduce the levels of seed or substrate. These diverse approaches include removal of aggregated asyn with passive or active immunization or by expression of vectorized antibodies, modulating kinetics of misfolding with small molecule anti-aggregants, lowering asyn gene expression by antisense oligonucleotides or inhibitory RNA, and pharmacological activation of asyn degradation pathways. We also discuss recent technological advances in combining low intensity focused ultrasound with intravenous microbubbles to transiently increase blood-brain barrier permeability for improved brain delivery and target engagement of these large molecule anti-asyn biologics.
نوع الوثيقة: article in journal/newspaper
اللغة: unknown
DOI: 10.3389/fneur.2022.852003
DOI: 10.3389/fneur.2022.852003/full
الإتاحة: https://doi.org/10.3389/fneur.2022.852003Test
حقوق: https://creativecommons.org/licenses/by/4.0Test/
رقم الانضمام: edsbas.9913469C
قاعدة البيانات: BASE