Protein production is an early biomarker for RNA ‐targeted therapies

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

(2018) Alex McCampbell et al.   JOURNAL OF CLINICAL INVESTIGATION

Tau Kinetics in Neurons and the Human Central Nervous System

(2018) Chihiro Sato et al.   NEURON

Development of a simple, rapid, and robust intrathecal catheterization method in the rat

(2017) Curt Mazur et al.   JOURNAL OF NEUROSCIENCE METHODS

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

(2017) Lindsay A. Becker et al.   NATURE

Age-Dependent Effects of apoE Reduction Using Antisense Oligonucleotides in a Model of β-amyloidosis

(2017) Tien-Phat V. Huynh et al.   NEURON

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tau reduction prevents neuronal loss and reverses pathological tau deposition and seeding in mice with tauopathy

(2017) Sarah L. DeVos et al.   Science Translational Medicine

Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72 -associated amyotrophic lateral sclerosis

(2017) Tania F. Gendron et al.   Science Translational Medicine

ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment?

(2017) Dmitry Petrov et al.   Frontiers in Aging Neuroscience

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

Defining SOD1 ALS natural history to guide therapeutic clinical trial design

(2016) Taha Bali et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Increased 4R-Tau Induces Pathological Changes in a Human-Tau Mouse Model

(2016) Kathleen M. Schoch et al.   NEURON

In vivo kinetic approach reveals slow SOD1 turnover in the CNS

(2015) Matthew J. Crisp et al.   JOURNAL OF CLINICAL INVESTIGATION

Preventing proteostasis diseases by selective inhibition of a phosphatase regulatory subunit

(2015) I. Das et al.   SCIENCE

Nerve Growth Factor Gene Therapy

(2015) Mark H. Tuszynski et al.   JAMA Neurology

Clinical trials in amyotrophic lateral sclerosis: why so many negative trials and how can trials be improved?

(2014) Hiroshi Mitsumoto et al.   LANCET NEUROLOGY

Alzheimer’s disease drug-development pipeline: few candidates, frequent failures

(2014) Jeffrey L Cummings et al.   Alzheimers Research & Therapy

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention

(2013) Christopher J. Donnelly et al.   NEURON

Antisense Oligonucleotides: Treating Neurodegeneration at the Level of RNA

(2013) Sarah L. DeVos et al.   Neurotherapeutics

SOD1 in Cerebral Spinal Fluid as a Pharmacodynamic Marker for Antisense Oligonucleotide Therapy

(2012) Leah Winer et al.   JAMA Neurology

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Hydrogen-deuterium exchange in vivo to measure turnover of an ALS-associated mutant SOD1 protein in spinal cord of mice

(2011) George W. Farr et al.   PROTEIN SCIENCE

Enhancement of proteasome activity by a small-molecule inhibitor of USP14

(2010) Byung-Hoon Lee et al.   NATURE

A γ-secretase inhibitor decreases amyloid-β production in the central nervous system

(2009) Randall J. Bateman et al.   ANNALS OF NEUROLOGY

Five siRNAs Targeting Three SNPs May Provide Therapy for Three-Quarters of Huntington's Disease Patients

(2009) Edith L. Pfister et al.   CURRENT BIOLOGY