Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era

Precision medicine in Parkinson’s disease: emerging treatments for genetic Parkinson’s disease

(2020) Susanne A. Schneider et al.   JOURNAL OF NEUROLOGY

Gold Nanoparticles in Conjunction with Nucleic Acids as a Modern Molecular System for Cellular Delivery

(2020) Anna Graczyk et al.   MOLECULES

Transcriptomic Profiling of Circular RNA in Different Brain Regions of Parkinson’s Disease in a Mouse Model

(2020) Erteng Jia et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Reversing a model of Parkinson’s disease with in situ converted nigral neurons

(2020) Hao Qian et al.   NATURE

LRRK2 impairs PINK1/Parkin-dependent mitophagy via its kinase activity: pathologic insights into Parkinson’s disease

(2019) Fiona Bonello et al.   HUMAN MOLECULAR GENETICS

Contributions of VPS35 mutations to Parkinson’s Disease

(2019) Abir A. Rahman et al.   NEUROSCIENCE

Treating Disease at the RNA Level with Oligonucleotides

(2019) Arthur A. Levin   NEW ENGLAND JOURNAL OF MEDICINE

Theranostic Strategy of Focused Ultrasound Induced Blood-Brain Barrier Opening for CNS Disease Treatment

(2019) Ko-Ting Chen et al.   Frontiers in Pharmacology

Role of UCHL1 in axonal injury and functional recovery after cerebral ischemia

(2019) Hao Liu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

PARP Inhibitors and Parkinson’s Disease

(2019) Abby L. Olsen et al.   NEW ENGLAND JOURNAL OF MEDICINE

Emerging links between pediatric lysosomal storage diseases and adult parkinsonism

(2019) Daniel Ysselstein et al.   MOVEMENT DISORDERS

Prevalence and duration of non-motor symptoms in prodromal Parkinson's disease

(2019) R. Durcan et al.   EUROPEAN JOURNAL OF NEUROLOGY

Targeting RNA : A Transformative Therapeutic Strategy

(2019) Wei Yin et al.   CTS-Clinical and Translational Science

A phase 0 first-in-human study using NU-0129: A gold base spherical nucleic acid (SNA) nanoconjugate targeting BCL2L12 in recurrent glioblastoma patients.

(2019) Priya Kumthekar et al.   JOURNAL OF CLINICAL ONCOLOGY

MicroRNA based theranostics for brain cancer: basic principles

(2019) George E. D. Petrescu et al.   JOURNAL OF EXPERIMENTAL & CLINICAL CANCER RESEARCH

Current Development of siRNA Bioconjugates: From Research to the Clinic

(2019) Ivan V. Chernikov et al.   Frontiers in Pharmacology

Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models

(2019) Youngsoo Kim et al.   MOLECULAR THERAPY

Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson’s disease

(2019) Takuya Uehara et al.   Scientific Reports

Progress in the genetic analysis of Parkinson’s disease

(2019) Andrew Singleton et al.   HUMAN MOLECULAR GENETICS

The genetic architecture of Parkinson's disease

(2019) Cornelis Blauwendraat et al.   LANCET NEUROLOGY

Genomewide association study of Parkinson's disease clinical biomarkers in 12 longitudinal patients' cohorts

(2019) Hirotaka Iwaki et al.   MOVEMENT DISORDERS

siRNA-Finder (si-Fi) Software for RNAi-Target Design and Off-Target Prediction

(2019) Stefanie Lück et al.   Frontiers in Plant Science

Drug Regulation in the Era of Individualized Therapies

(2019) Janet Woodcock et al.   NEW ENGLAND JOURNAL OF MEDICINE

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

(2019) Jinkuk Kim et al.   NEW ENGLAND JOURNAL OF MEDICINE

DJ-1 regulates the integrity and function of ER-mitochondria association through interaction with IP3R3-Grp75-VDAC1

(2019) Yi Liu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Overcoming the delivery barrier of oligonucleotide drugs and enhancing nucleoside drug efficiency: The use of nucleolipids

(2019) Xinyang Zhou et al.   MEDICINAL RESEARCH REVIEWS

Salivary microR‐153 and microR‐223 Levels as Potential Diagnostic Biomarkers of Idiopathic Parkinson's Disease

(2019) Marisa Cressatti et al.   MOVEMENT DISORDERS

Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides

(2019) Juergen Scharner et al.   NUCLEIC ACIDS RESEARCH

Applications of Spherical Nucleic Acid Nanoparticles as Delivery Systems

(2019) Ahad Mokhtarzadeh et al.   TRENDS IN MOLECULAR MEDICINE

Alpha-synuclein delays mitophagy and targeting Miro rescues neuron loss in Parkinson’s models

(2018) Atossa Shaltouki et al.   ACTA NEUROPATHOLOGICA

Nucleic acids delivering nucleic acids

(2018) Silvia Catuogno et al.   ADVANCED DRUG DELIVERY REVIEWS

Ubiquitin C-Terminal Hydrolase L1 regulates autophagy by inhibiting autophagosome formation through its deubiquitinating enzyme activity

(2018) Cong Yan et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Th17 Lymphocytes Induce Neuronal Cell Death in a Human iPSC-Based Model of Parkinson’s Disease

(2018) Annika Sommer et al.   Cell Stem Cell

Mitochondrial Dysfunction in Parkinson’s Disease: New Mechanistic Insights and Therapeutic Perspectives

(2018) Jin-Sung Park et al.   Current Neurology and Neuroscience Reports

Parkinson's disease-related DJ-1 modulates the expression of uncoupling protein 4 against oxidative stress

(2018) Shaoqing Xu et al.   JOURNAL OF NEUROCHEMISTRY

A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II

(2018) Hiroyuki Sonoda et al.   MOLECULAR THERAPY

Selective α-Synuclein Knockdown in Monoamine Neurons by Intranasal Oligonucleotide Delivery: Potential Therapy for Parkinson’s Disease

(2018) Diana Alarcón-Arís et al.   MOLECULAR THERAPY

Eteplirsen treatment for Duchenne muscular dystrophy

(2018) Jay S. Charleston et al.   NEUROLOGY

Mitochondrial calcium imbalance in Parkinson’s disease

(2018) Marthe H.R. Ludtmann et al.   NEUROSCIENCE LETTERS

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Identifying and avoiding off-target effects of RNase H-dependent antisense oligonucleotides in mice

(2018) Peter H Hagedorn et al.   NUCLEIC ACIDS RESEARCH

Regulation of myeloid cell phagocytosis by LRRK2 via WAVE2 complex stabilization is altered in Parkinson’s disease

(2018) Kwang Soo Kim et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification of a highly neurotoxic α-synuclein species inducing mitochondrial damage and mitophagy in Parkinson’s disease

(2018) Diego Grassi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

LRRK2 kinase in Parkinson's disease

(2018) Dario R. Alessi et al.   SCIENCE

ER–mitochondria signaling in Parkinson’s disease

(2018) Patricia Gómez-Suaga et al.   Cell Death & Disease

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

(2018) Maja M. Janas et al.   Nature Communications

Blood–brain barrier opening in Alzheimer’s disease using MR-guided focused ultrasound

(2018) Nir Lipsman et al.   Nature Communications

Intracerebroventricular Administration of a 2′-O-Methyl Phosphorothioate Antisense Oligonucleotide Results in Activation of the Innate Immune System in Mouse Brain

(2018) Lodewijk J.A. Toonen et al.   Nucleic Acid Therapeutics

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

(2018) Aaron D. Springer et al.   Nucleic Acid Therapeutics

Lack of PINK1 alters glia innate immune responses and enhances inflammation-induced, nitric oxide-mediated neuron death

(2018) Liuke Sun et al.   Scientific Reports

Progression in the LRRK2-Associated Parkinson Disease Population

(2018) Rachel Saunders-Pullman et al.   JAMA Neurology

Distinct Mechanisms of Pathogenic DJ-1 Mutations in Mitochondrial Quality Control

(2018) Daniela Strobbe et al.   Frontiers in Molecular Neuroscience

Circular siRNAs for Reducing Off-Target Effects and Enhancing Long-Term Gene Silencing in Cells and Mice

(2018) Liangliang Zhang et al.   Molecular Therapy-Nucleic Acids

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

(2018) Loren L. Flynn et al.   Molecular Therapy-Nucleic Acids

Novel DNA Aptamers for Parkinson’s Disease Treatment Inhibit α-Synuclein Aggregation and Facilitate its Degradation

(2018) Yuan Zheng et al.   Molecular Therapy-Nucleic Acids

Microglia and Beyond: Innate Immune Cells As Regulators of Brain Development and Behavioral Function

(2018) Kathryn M. Lenz et al.   Frontiers in Immunology

Neuroinflammation: Microglia and T Cells Get Ready to Tango

(2018) Sjoerd T. T. Schetters et al.   Frontiers in Immunology

Cytoprotective mechanisms of DJ-1 against oxidative stress through modulating ERK1/2 and ASK1 signal transduction

(2018) Stephanie E. Oh et al.   Redox Biology

Mitochondrial Dysfunction and Mitophagy Defect Triggered by Heterozygous GBA Mutations

(2018) Hongyu Li et al.   Autophagy

Peripheral Administration of Antisense Oligonucleotides Targeting the Amyloid-β Protein Precursor Reverses AβPP and LRP-1 Overexpression in the Aged SAMP8 Mouse Brain

(2018) Michelle A. Erickson et al.   JOURNAL OF ALZHEIMERS DISEASE

Pink1/Parkin link inflammation, mitochondrial stress, and neurodegeneration

(2018) Laura E. Newman et al.   JOURNAL OF CELL BIOLOGY

Targeting of the class II transactivator attenuates inflammation and neurodegeneration in an alpha-synuclein model of Parkinson’s disease

(2018) Gregory P. Williams et al.   Journal of Neuroinflammation

Immunotherapy targeting toll-like receptor 2 alleviates neurodegeneration in models of synucleinopathy by modulating α-synuclein transmission and neuroinflammation

(2018) Changyoun Kim et al.   Molecular Neurodegeneration

Mitochondrial double-stranded RNA triggers antiviral signalling in humans

(2018) Ashish Dhir et al.   NATURE

Gene-silencing technology gets first drug approval after 20-year wait

(2018) Heidi Ledford   NATURE

Parkin and PINK1 mitigate STING-induced inflammation

(2018) Danielle A. Sliter et al.   NATURE

The role of T cells in the pathogenesis of Parkinson’s disease

(2018) Zhichun Chen et al.   PROGRESS IN NEUROBIOLOGY

Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping

(2018) Dunhui Li et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

Chemical Modification Study of Antisense Gapmers

(2018) Robert Stanton et al.   Nucleic Acid Therapeutics

Atypical parkinsonism of progressive supranuclear palsy–parkinsonism (PSP-P) phenotype with rare variants in FBXO7 and VPS35 genes associated with Lewy body pathology

(2018) Kateřina Menšíková et al.   ACTA NEUROPATHOLOGICA

Three decades of nucleic acid aptamer technologies: Lessons learned, progress and opportunities on aptamer development

(2018) Tao Wang et al.   BIOTECHNOLOGY ADVANCES

ATP13A2 facilitates HDAC6 recruitment to lysosome to promote autophagosome–lysosome fusion

(2018) Ruoxi Wang et al.   JOURNAL OF CELL BIOLOGY

Pathogenesis-targeted therapeutic strategies in Parkinson's disease

(2018) Joseph Jankovic   MOVEMENT DISORDERS

Genotype-phenotype relations for the Parkinson's disease genes SNCA, LRRK2, VPS35: MDSGene systematic review

(2018) Joanne Trinh et al.   MOVEMENT DISORDERS

Cell-Penetrating Peptide Conjugates of Steric Blocking Oligonucleotides as Therapeutics for Neuromuscular Diseases from a Historical Perspective to Current Prospects of Treatment

(2018) Michael J. Gait et al.   Nucleic Acid Therapeutics

SLP-2 interacts with Parkin in mitochondria and prevents mitochondrial dysfunction in Parkin-deficient human iPSC-derived neurons and Drosophila

(2017) Alessandra Zanon et al.   HUMAN MOLECULAR GENETICS

ATP13A2/PARK9 regulates endo-/lysosomal cargo sorting and proteostasis through a novel PI(3, 5)P2-mediated scaffolding function

(2017) S. Demirsoy et al.   HUMAN MOLECULAR GENETICS

microRNAs in Parkinson’s Disease: From Pathogenesis to Novel Diagnostic and Therapeutic Approaches

(2017) Loredana Leggio et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

LRRK2 promotes the activation of NLRC4 inflammasome duringSalmonellaTyphimurium infection

(2017) Weiwei Liu et al.   JOURNAL OF EXPERIMENTAL MEDICINE

Plasma and White Blood Cells Show Different miRNA Expression Profiles in Parkinson’s Disease

(2017) Christine Schwienbacher et al.   JOURNAL OF MOLECULAR NEUROSCIENCE

Mechanistic contributions of FBXO7 to Parkinson disease

(2017) Sabitha Joseph et al.   JOURNAL OF NEUROCHEMISTRY

Mitochondrial Calcium Dysregulation Contributes to Dendrite Degeneration Mediated by PD/LBD-Associated LRRK2 Mutants

(2017) Manish Verma et al.   JOURNAL OF NEUROSCIENCE

The Trojan horse - neuroinflammatory impact of T cells in neurodegenerative diseases

(2017) Annika Sommer et al.   Molecular Neurodegeneration

Loss of MicroRNA-7 Regulation Leads to α-Synuclein Accumulation and Dopaminergic Neuronal Loss In Vivo

(2017) Kirsty J. McMillan et al.   MOLECULAR THERAPY

FDA-Approved Oligonucleotide Therapies in 2017

(2017) Cy A. Stein et al.   MOLECULAR THERAPY

Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease

(2017) Valentina Sardone et al.   MOLECULES

“Miro” in Parkinson's disease: Here, there, everywhere!

(2017) Abhishek Lenka et al.   MOVEMENT DISORDERS

Anionic liposomes for small interfering ribonucleic acid (siRNA) delivery to primary neuronal cells: Evaluation of alpha-synuclein knockdown efficacy

(2017) Michele Schlich et al.   Nano Research

Oligonucleotide therapies for disorders of the nervous system

(2017) Olga Khorkova et al.   NATURE BIOTECHNOLOGY

MicroRNA therapeutics: towards a new era for the management of cancer and other diseases

(2017) Rajesha Rupaimoole et al.   NATURE REVIEWS DRUG DISCOVERY

Recent findings on the physiological function of DJ-1: Beyond Parkinson's disease

(2017) Alice Biosa et al.   NEUROBIOLOGY OF DISEASE

Increased abundance of myeloid-derived suppressor cells and Th17 cells in peripheral blood of newly-diagnosed Parkinson’s disease patients

(2017) Siyuan Chen et al.   NEUROSCIENCE LETTERS

Managing the sequence-specificity of antisense oligonucleotides in drug discovery

(2017) Peter H. Hagedorn et al.   NUCLEIC ACIDS RESEARCH

Intracerebroventricular Delivery as a Safe, Long-Term Route of Drug Administration

(2017) Jessica L. Cohen-Pfeffer et al.   PEDIATRIC NEUROLOGY

The Yin and Yang of nucleic acid-based therapy in the brain

(2017) Stefano Gustincich et al.   PROGRESS IN NEUROBIOLOGY

Pathological α-synuclein exacerbates the progression of Parkinson’s disease through microglial activation

(2017) Qiu-Shuang Zhang et al.   TOXICOLOGY LETTERS

PINK1-mediated phosphorylation of LETM1 regulates mitochondrial calcium transport and protects neurons against mitochondrial stress

(2017) En Huang et al.   Nature Communications

Distribution and Penetration of Intracerebroventricularly Administered 2′OMePS Oligonucleotide in the Mouse Brain

(2017) João Casaca-Carreira et al.   Nucleic Acid Therapeutics

Inhibition by Multifunctional Magnetic Nanoparticles Loaded with Alpha-Synuclein RNAi Plasmid in a Parkinson's Disease Model

(2017) Shuiqin Niu et al.   Theranostics

PINK1 Primes Parkin-Mediated Ubiquitination of PARIS in Dopaminergic Neuronal Survival

(2017) Yunjong Lee et al.   Cell Reports

MicroRNA-4639 Is a Regulator of DJ-1 Expression and a Potential Early Diagnostic Marker for Parkinson’s Disease

(2017) Yimeng Chen et al.   Frontiers in Aging Neuroscience

Targeting Microglial Activation States as a Therapeutic Avenue in Parkinson’s Disease

(2017) Sudhakar R. Subramaniam et al.   Frontiers in Aging Neuroscience

PINK1, Parkin, and Mitochondrial Quality Control: What can we Learn about Parkinson’s Disease Pathobiology?

(2017) Dominika Truban et al.   Journal of Parkinsons Disease

PINK1/Parkin-Dependent Mitochondrial Surveillance: From Pleiotropy to Parkinson's Disease

(2017) Francois Mouton-Liger et al.   Frontiers in Molecular Neuroscience

Polyethylenimine Nanoparticle-Mediated siRNA Delivery to Reduce α-Synuclein Expression in a Model of Parkinson’s Disease

(2017) Christin Helmschrodt et al.   Molecular Therapy-Nucleic Acids

Exosome Mediated Delivery of miR-124 Promotes Neurogenesis after Ischemia

(2017) Jialei Yang et al.   Molecular Therapy-Nucleic Acids

Role of Cell-Penetrating Peptides in Intracellular Delivery of Peptide Nucleic Acids Targeting Hepadnaviral Replication

(2017) Bénédicte Ndeboko et al.   Molecular Therapy-Nucleic Acids

Therapeutic miRNA and siRNA: Moving from Bench to Clinic as Next Generation Medicine

(2017) Chiranjib Chakraborty et al.   Molecular Therapy-Nucleic Acids

LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model

(2017) Hien Tran Zhao et al.   Molecular Therapy-Nucleic Acids

Mitophagy in Parkinson’s Disease: Pathogenic and Therapeutic Implications

(2017) Fei Gao et al.   Frontiers in Neurology

Parkinson disease

(2017) Werner Poewe et al.   Nature Reviews Disease Primers

Proteasome impairment by α-synuclein

(2017) Lisa Zondler et al.   PLoS One

Delivery of Small Interfering RNA to Inhibit Vascular Endothelial Growth Factor in Zebrafish Using Natural Brain Endothelia Cell-Secreted Exosome Nanovesicles for the Treatment of Brain Cancer

(2016) Tianzhi Yang et al.   AAPS Journal

Pharmacokinetics and biodistribution of recently-developed siRNA nanomedicines

(2016) Jinho Park et al.   ADVANCED DRUG DELIVERY REVIEWS

A Novel Nonsense Mutation in DNAJC 6 Expands the Phenotype of Autosomal-Recessive Juvenile-Onset Parkinson's Disease

(2016) Liena Elbaghir Omer Elsayed et al.   ANNALS OF NEUROLOGY

D NAJC 6 Mutations Associated With Early-Onset Parkinson's Disease

(2016) Simone Olgiati et al.   ANNALS OF NEUROLOGY

Regulation of DJ-1 by Glutaredoxin 1 in Vivo: Implications for Parkinson’s Disease

(2016) William M. Johnson et al.   BIOCHEMISTRY

Functional Impairment in Miro Degradation and Mitophagy Is a Shared Feature in Familial and Sporadic Parkinson’s Disease

(2016) Chung-Han Hsieh et al.   Cell Stem Cell

Aptamer and its applications in neurodegenerative diseases

(2016) Jing Qu et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Evaluation of anhydrohexitol nucleic acid, cyclohexenyl nucleic acid and d-altritol nucleic acid-modified 2′-O-methyl RNA mixmer antisense oligonucleotides for exon skipping in vitro

(2016) Bao T. Le et al.   CHEMICAL COMMUNICATIONS

Nurr1-Based Therapies for Parkinson's Disease

(2016) Jie Dong et al.   CNS Neuroscience & Therapeutics

Inhibition of the mitochondrial calcium uniporter rescues dopaminergic neurons inpink1−/−zebrafish

(2016) Smijin Soman et al.   EUROPEAN JOURNAL OF NEUROSCIENCE

N-Adamantyl-4-methylthiazol-2-amine suppresses amyloid β-induced neuronal oxidative damage in cortical neurons

(2016) Chang Hun Cho et al.   FREE RADICAL RESEARCH

Oxidant-induced Interprotein Disulfide Formation in Cardiac Protein DJ-1 Occurs via an Interaction with Peroxiredoxin 2

(2016) Mariana Fernandez-Caggiano et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Intrathecal Injections in Children With Spinal Muscular Atrophy

(2016) Manon Haché et al.   JOURNAL OF CHILD NEUROLOGY

MicroRNA-124 loaded nanoparticles enhance brain repair in Parkinson's disease

(2016) C. Saraiva et al.   JOURNAL OF CONTROLLED RELEASE

Infiltrating T lymphocytes reduce myeloid phagocytosis activity in synucleinopathy model

(2016) Annika Sommer et al.   Journal of Neuroinflammation

PINK1 in the limelight: multiple functions of an eclectic protein in human health and disease

(2016) Giuseppe Arena et al.   JOURNAL OF PATHOLOGY

DJ-1/PARK7, But Not Its L166P Mutant Linked to Autosomal Recessive Parkinsonism, Modulates the Transcriptional Activity of the Orphan Nuclear Receptor Nurr1 In Vitro and In Vivo

(2016) Lingling Lu et al.   MOLECULAR NEUROBIOLOGY

Th17 Cells Induce Dopaminergic Neuronal Death via LFA-1/ICAM-1 Interaction in a Mouse Model of Parkinson’s Disease

(2016) Zhan Liu et al.   MOLECULAR NEUROBIOLOGY

MicroRNA-7 targets Nod-like receptor protein 3 inflammasome to modulate neuroinflammation in the pathogenesis of Parkinson’s disease

(2016) Yan Zhou et al.   Molecular Neurodegeneration

Shp-2 knockdown preventsl-dopa-induced dyskinesia in a rat model of Parkinson's disease

(2016) Chiara Fiorentini et al.   MOVEMENT DISORDERS

Autophagy and Alpha-Synuclein: Relevance to Parkinson's Disease and Related Synucleopathies

(2016) Maria Xilouri et al.   MOVEMENT DISORDERS

Identification of TMEM230 mutations in familial Parkinson's disease

(2016) Han-Xiang Deng et al.   NATURE GENETICS

From blood–brain barrier to blood–brain interface: new opportunities for CNS drug delivery

(2016) William A. Banks   NATURE REVIEWS DRUG DISCOVERY

Aptamers as targeted therapeutics: current potential and challenges

(2016) Jiehua Zhou et al.   NATURE REVIEWS DRUG DISCOVERY

Inhibition of microRNA-210 provides neuroprotection in hypoxic–ischemic brain injury in neonatal rats

(2016) Qingyi Ma et al.   NEUROBIOLOGY OF DISEASE

Astrocytes: Integrative Regulators of Neuroinflammation in Stroke and Other Neurological Diseases

(2016) Egle Cekanaviciute et al.   Neurotherapeutics

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

Challenges in detecting disease modification in Parkinson's disease clinical trials

(2016) Dilan Athauda et al.   PARKINSONISM & RELATED DISORDERS

Exosomes as a potential novel therapeutic tools against neurodegenerative diseases

(2016) Akvilė Jarmalavičiūtė et al.   PHARMACOLOGICAL RESEARCH

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

(2016) Suzan M. Hammond et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Caspase-1 causes truncation and aggregation of the Parkinson’s disease-associated protein α-synuclein

(2016) Wei Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Oxidative stress: A major pathogenesis and potential therapeutic target of antioxidative agents in Parkinson’s disease and Alzheimer’s disease

(2016) Tianfang Jiang et al.   PROGRESS IN NEUROBIOLOGY

α-Synuclein binds to TOM20 and inhibits mitochondrial protein import in Parkinson’s disease

(2016) Roberto Di Maio et al.   Science Translational Medicine

Deciphering the Molecular Signals of PINK1/Parkin Mitophagy

(2016) Thanh N. Nguyen et al.   TRENDS IN CELL BIOLOGY

Effects of Repeated Complement Activation Associated with Chronic Treatment of Cynomolgus Monkeys with 2′-O-Methoxyethyl Modified Antisense Oligonucleotide

(2016) Lijiang Shen et al.   Nucleic Acid Therapeutics

Investigation of twisted intercalating nucleic acid (TINA)-modified antisense oligonucleotides for splice modulation by induced exon-skipping in vitro

(2016) Bao T. Le et al.   RSC Advances

Exosome-mediated small RNA delivery for gene therapy

(2016) Yu Zhou et al.   Wiley Interdisciplinary Reviews-RNA

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Delivery of therapeutic oligonucleotides with cell penetrating peptides

(2015) Prisca Boisguérin et al.   ADVANCED DRUG DELIVERY REVIEWS

Ubiquitin C-terminal hydrolases cleave isopeptide- and peptide-linked ubiquitin from structured proteins but do not edit ubiquitin homopolymers

(2015) John S. Bett et al.   BIOCHEMICAL JOURNAL

MicroRNA-214 participates in the neuroprotective effect of Resveratrol via inhibiting α-synuclein expression in MPTP-induced Parkinson’s disease mouse

(2015) Zhao-Hui Wang et al.   BIOMEDICINE & PHARMACOTHERAPY

Role of Nuclear Factor Erythroid 2–Related Factor 2 in the Oxidative Stress–Dependent Hypertension Associated With the Depletion of DJ-1Novelty and Significance

(2015) Santiago Cuevas et al.   HYPERTENSION

Epidermal Growth Factor-dependent Activation of the Extracellular Signal-regulated Kinase Pathway by DJ-1 Protein through Its Direct Binding to c-Raf Protein

(2015) Kazuko Takahashi-Niki et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Leucine-rich Repeat Kinase 2 (LRRK2) Pharmacological Inhibition Abates α-Synuclein Gene-induced Neurodegeneration

(2015) João P. L. Daher et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

shRNA targeting α-synuclein prevents neurodegeneration in a Parkinson’s disease model

(2015) Alevtina D. Zharikov et al.   JOURNAL OF CLINICAL INVESTIGATION

New insights on Parkinson’s disease genes: the link between mitochondria impairment and neuroinflammation

(2015) Dorit Trudler et al.   JOURNAL OF NEURAL TRANSMISSION

CHCHD2 mutations in autosomal dominant late-onset Parkinson's disease: a genome-wide linkage and sequencing study

(2015) Manabu Funayama et al.   LANCET NEUROLOGY

Therapeutic antidepressant potential of a conjugated siRNA silencing the serotonin transporter after intranasal administration

(2015) A Ferrés-Coy et al.   MOLECULAR PSYCHIATRY

709. rAAV-Mediated Regulation of Striatal Nurr1 Expression Alters Development and Severity of Levodopa-Induced Dyskinesias in the 6-OHDA Rat Model of Parkinson's Disease

(2015) Rhyomi C. Sellnow et al.   MOLECULAR THERAPY

Knocking down disease: a progress report on siRNA therapeutics

(2015) Anders Wittrup et al.   NATURE REVIEWS GENETICS

The relation between α-synuclein and microglia in Parkinson’s disease: Recent developments

(2015) V. Sanchez-Guajardo et al.   NEUROSCIENCE

The isolation of an RNA aptamer targeting to p53 protein with single amino acid mutation

(2015) Liang Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Increased brain uptake of targeted nanoparticles by adding an acid-cleavable linkage between transferrin and the nanoparticle core

(2015) Andrew J. Clark et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Mitochondrial dysfunction and mitophagy in Parkinson's: from familial to sporadic disease

(2015) Brent J. Ryan et al.   TRENDS IN BIOCHEMICAL SCIENCES

Insights into Neuroinflammation in Parkinson’s Disease: From Biomarkers to Anti-Inflammatory Based Therapies

(2015) Natália Pessoa Rocha et al.   Biomed Research International

Normalization of Overexpressed α-Synuclein Causing Parkinson's Disease By a Moderate Gene Silencing With RNA Interference

(2015) Masaki Takahashi et al.   Molecular Therapy-Nucleic Acids

Neuroinflammation in Parkinson’s disease and its potential as therapeutic target

(2015) Qinqin Wang et al.   Translational Neurodegeneration

Enhancement of Blood–Brain Barrier Permeability and Delivery of Antisense Oligonucleotides or Plasmid DNA to the Brain by the Combination of Bubble Liposomes and High-Intensity Focused Ultrasound

(2015) Yoichi Negishi et al.   Pharmaceutics

Poly (ADP-ribose) in the pathogenesis of Parkinson's disease

(2014) Yunjong Lee et al.   BMB Reports

Ubiquitin code assembly and disassembly

(2014) Claire Heride et al.   CURRENT BIOLOGY

MicroRNA Therapeutics in Neurological Disease

(2014) David Greenberg et al.   CURRENT PHARMACEUTICAL DESIGN

Aptamer-based therapeutics of the past, present and future: from the perspective of eye-related diseases

(2014) Jagat R. Kanwar et al.   DRUG DISCOVERY TODAY

LETM1-dependent mitochondrial Ca2+ flux modulates cellular bioenergetics and proliferation

(2014) Patrick J. Doonan et al.   FASEB JOURNAL

MicroRNA-7a regulates pancreatic β cell function

(2014) Mathieu Latreille et al.   JOURNAL OF CLINICAL INVESTIGATION

VPS35 Parkinson’s disease phenotype resembles the sporadic disease

(2014) Walter Struhal et al.   JOURNAL OF NEURAL TRANSMISSION

Ultrasensitive and selective voltammetric aptasensor for dopamine based on a conducting polymer nanocomposite doped with graphene oxide

(2014) Wenting Wang et al.   MICROCHIMICA ACTA

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

(2014) Amber L Southwell et al.   MOLECULAR THERAPY

Systemic exosomal siRNA delivery reduced alpha-synuclein aggregates in brains of transgenic mice

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