Progress towards therapies for disease modification in Parkinson's disease

N Vijiaratnam, T Simuni, O Bandmann… - The Lancet …, 2021 - thelancet.com
The development of interventions to slow or halt the progression of Parkinson's disease
remains a priority for patients and researchers alike. To date, no agents have been shown to …

GBA Variants and Parkinson Disease: Mechanisms and Treatments

L Smith, AHV Schapira - Cells, 2022 - mdpi.com
The GBA gene encodes for the lysosomal enzyme glucocerebrosidase (GCase), which
maintains glycosphingolipid homeostasis. Approximately 5–15% of PD patients have …

Gut-seeded α-synuclein fibrils promote gut dysfunction and brain pathology specifically in aged mice

C Challis, A Hori, TR Sampson, BB Yoo… - Nature …, 2020 - nature.com
Parkinson's disease is a synucleinopathy that is characterized by motor dysfunction, death of
midbrain dopaminergic neurons and accumulation of α-synuclein (α-Syn) aggregates …

Modeling Parkinson's disease with the alpha-synuclein protein

M Gómez-Benito, N Granado, P García-Sanz… - Frontiers in …, 2020 - frontiersin.org
Alpha-synuclein (α-Syn) is a key protein involved in Parkinson's disease (PD) pathology. PD
is characterized by the loss of dopaminergic neuronal cells in the substantia nigra pars …

α-synuclein toxicity in neurodegeneration: mechanism and therapeutic strategies

YC Wong, D Krainc - Nature medicine, 2017 - nature.com
Alterations in α-synuclein dosage lead to familial Parkinson's disease (PD), and its
accumulation results in synucleinopathies that include PD, dementia with Lewy bodies …

Gene therapy for neurological disorders: progress and prospects

BE Deverman, BM Ravina, KS Bankiewicz… - Nature Reviews Drug …, 2018 - nature.com
Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

GBA, Gaucher Disease, and Parkinson's Disease: From Genetic to Clinic to New Therapeutic Approaches

GM Riboldi, AB Di Fonzo - Cells, 2019 - mdpi.com
Parkinson's disease (PD) is the second most common degenerative disorder. Although the
disease was described more than 200 years ago, its pathogenetic mechanisms have not yet …

Therapeutic potential of autophagy-enhancing agents in Parkinson's disease

TE Moors, JJM Hoozemans, A Ingrassia… - Molecular …, 2017 - Springer
Converging evidence from genetic, pathological and experimental studies have increasingly
suggested an important role for autophagy impairment in Parkinson's Disease (PD). Genetic …

Adeno-associated virus-based gene therapy for CNS diseases

M Hocquemiller, L Giersch, M Audrain… - Human gene …, 2016 - liebertpub.com
Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Activation of β-glucocerebrosidase reduces pathological α-synuclein and restores lysosomal function in Parkinson's patient midbrain neurons

JR Mazzulli, F Zunke, T Tsunemi, NJ Toker… - Journal of …, 2016 - Soc Neuroscience
Parkinson's disease (PD) is characterized by the accumulation of α-synuclein (α-syn) within
Lewy body inclusions in the nervous system. There are currently no disease-modifying …