Lista de obras -

A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS

scientific article published on 24 September 2020

A viral toolkit for recording transcription factor-DNA interactions in live mouse tissues

scientific article published on 16 April 2020

ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import.

artículo científico publicado en 2010

Amyotrophic Lateral Sclerosis Care and Research in the USA During the COVID-19 Pandemic: Challenges and Opportunities

artículo científico publicado en 2020

Amyotrophic lateral sclerosis and gene therapy.

artículo científico publicado en 2006

Amyotrophic lateral sclerosis onset is influenced by the burden of rare variants in known amyotrophic lateral sclerosis genes.

artículo científico publicado en 2014

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

scientific article published on 29 March 2013

An endogenous peptide marker differentiates SOD1 stability and facilitates pharmacodynamic monitoring in SOD1 amyotrophic lateral sclerosis

scientific article published on 16 May 2019

Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases

artículo científico publicado en 2017

Antisense oligonucleotide therapy for neurodegenerative disease.

artículo científico publicado en 2006

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

artículo científico publicado en 2018

Antisense reduction of tau in adult mice protects against seizures

artículo científico publicado en 2013

Burst mitofusin activation reverses neuromuscular dysfunction in murine CMT2A

artículo científico publicado en 2020

CK-2127107 amplifies skeletal muscle response to nerve activation in humans.

artículo científico publicado en 2017

CNS microRNA profiles: a database for cell type enriched microRNA expression across the mouse central nervous system

scientific article published on 18 March 2020

Familial ALS with extreme phenotypic variability due to the I113T SOD1 mutation

article

Fibroblast bioenergetics to classify amyotrophic lateral sclerosis patients.

artículo científico publicado en 2017

Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis

artículo científico publicado en 2006

Gene-targeted therapies for the central nervous system

artículo científico publicado en 2008

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene

artículo científico publicado en 2018

Has gene therapy for ALS arrived?

artículo científico publicado en 2003

Human genetics and neuropathology suggest a link between miR-218 and amyotrophic lateral sclerosis pathophysiology

artículo científico publicado en 2019

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

artículo científico publicado en 2016

Long-Term Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate-Taurursodiol in ALS

scientific article published on 16 October 2020

Management of amyotrophic lateral sclerosis

artículo científico publicado en 2013

Medicine. Treating neurodegenerative diseases with antibiotics.

artículo científico publicado en 2005

Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice

artículo científico publicado en 2013

MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.

artículo científico publicado en 2017

Motor neuron-derived microRNAs cause astrocyte dysfunction in amyotrophic lateral sclerosis

scientific article published on 01 September 2018

Peripheral neuropathy in an outpatient cohort of patients with Sjögren's syndrome

artículo científico publicado en 2006

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

artículo científico publicado en 2020

Phosphorylated neurofilament heavy chain: A biomarker of survival for C9ORF72-associated amyotrophic lateral sclerosis

artículo científico publicado en 2017

Plasma neurofilament light predicts mortality in patients with stroke

scientific article published on 01 November 2020

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

artículo científico

Pre-morbid type 2 diabetes mellitus is not a prognostic factor in amyotrophic lateral sclerosis

artículo científico publicado en 2015

Progressive spinal axonal degeneration and slowness in ALS2-deficient mice

artículo científico publicado en 2006

Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkers

scientific article published on 02 October 2019

RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia

artículo científico publicado en 2013

RCADiA: simple automation platform for comparative multidimensional protein identification technology

artículo científico publicado en 2007

Reply: To PMID 25900666

artículo científico publicado en 2015

SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy

scientific article published on February 2013

Selective association of misfolded ALS-linked mutant SOD1 with the cytoplasmic face of mitochondria.

artículo científico publicado en 2008

Smartphone data during the COVID-19 pandemic can quantify behavioral changes in people with ALS

artículo científico publicado en 2020

Synthesis and in vitro characterization of a P2X7 radioligand [123I]TZ6019 and its response to neuroinflammation in a mouse model of Alzheimer disease

artículo científico publicado en 2017

TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration

artículo científico publicado en 2009

TREM2 variant p.R47H as a risk factor for sporadic amyotrophic lateral sclerosis

artículo científico publicado en 2014

Toxicity of familial ALS-linked SOD1 mutants from selective recruitment to spinal mitochondria.

artículo científico publicado en 2004

Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis

scientific article published on 01 September 2020

Unexpected similarities between C9ORF72 and sporadic forms of ALS/FTD suggest a common disease mechanism

artículo científico publicado en 2018

Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis

artículo científico publicado en 2005

Lista de obras de

A Phase 2, Double-Blind, Randomized, Dose-Ranging Trial Of Reldesemtiv In Patients With ALS

A viral toolkit for recording transcription factor-DNA interactions in live mouse tissues

ALS-linked mutant superoxide dismutase 1 (SOD1) alters mitochondrial protein composition and decreases protein import.

Amyotrophic Lateral Sclerosis Care and Research in the USA During the COVID-19 Pandemic: Challenges and Opportunities

Amyotrophic lateral sclerosis and gene therapy.

Amyotrophic lateral sclerosis onset is influenced by the burden of rare variants in known amyotrophic lateral sclerosis genes.

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

An endogenous peptide marker differentiates SOD1 stability and facilitates pharmacodynamic monitoring in SOD1 amyotrophic lateral sclerosis

Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases

Antisense oligonucleotide therapy for neurodegenerative disease.

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

Antisense reduction of tau in adult mice protects against seizures

Burst mitofusin activation reverses neuromuscular dysfunction in murine CMT2A

CK-2127107 amplifies skeletal muscle response to nerve activation in humans.

CNS microRNA profiles: a database for cell type enriched microRNA expression across the mouse central nervous system

Familial ALS with extreme phenotypic variability due to the I113T SOD1 mutation

Fibroblast bioenergetics to classify amyotrophic lateral sclerosis patients.

Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis

Gene-targeted therapies for the central nervous system

Genome-wide Analyses Identify KIF5A as a Novel ALS Gene

Has gene therapy for ALS arrived?

Human genetics and neuropathology suggest a link between miR-218 and amyotrophic lateral sclerosis pathophysiology

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

Long-Term Survival of Participants in the CENTAUR Trial of Sodium Phenylbutyrate-Taurursodiol in ALS

Management of amyotrophic lateral sclerosis

Medicine. Treating neurodegenerative diseases with antibiotics.

Method for widespread microRNA-155 inhibition prolongs survival in ALS-model mice

MicroRNA Profiling Reveals Marker of Motor Neuron Disease in ALS Models.

Motor neuron-derived microRNAs cause astrocyte dysfunction in amyotrophic lateral sclerosis

Peripheral neuropathy in an outpatient cohort of patients with Sjögren's syndrome

Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

Phosphorylated neurofilament heavy chain: A biomarker of survival for C9ORF72-associated amyotrophic lateral sclerosis

Plasma neurofilament light predicts mortality in patients with stroke

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

Pre-morbid type 2 diabetes mellitus is not a prognostic factor in amyotrophic lateral sclerosis

Progressive spinal axonal degeneration and slowness in ALS2-deficient mice

Prospective natural history study of C9orf72 ALS clinical characteristics and biomarkers

RAN proteins and RNA foci from antisense transcripts in C9ORF72 ALS and frontotemporal dementia

RCADiA: simple automation platform for comparative multidimensional protein identification technology

Reply: To PMID 25900666

SOD1 in cerebral spinal fluid as a pharmacodynamic marker for antisense oligonucleotide therapy

Selective association of misfolded ALS-linked mutant SOD1 with the cytoplasmic face of mitochondria.

Smartphone data during the COVID-19 pandemic can quantify behavioral changes in people with ALS

Synthesis and in vitro characterization of a P2X7 radioligand [123I]TZ6019 and its response to neuroinflammation in a mouse model of Alzheimer disease

TDP-43 mutant transgenic mice develop features of ALS and frontotemporal lobar degeneration

TREM2 variant p.R47H as a risk factor for sporadic amyotrophic lateral sclerosis

Toxicity of familial ALS-linked SOD1 mutants from selective recruitment to spinal mitochondria.

Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis

Unexpected similarities between C9ORF72 and sporadic forms of ALS/FTD suggest a common disease mechanism

Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis