Developments in treatments for amyotrophic lateral sclerosis via intracerebroventricular or intrathecal delivery

Therapeutic potentials of gene silencing by RNA interference: Principles, challenges, and new strategies

(2014) Yan Deng et al.   GENE

Adeno-associated Virus–mediated Delivery of a Recombinant Single-chain Antibody Against Misfolded Superoxide Dismutase for Treatment of Amyotrophic Lateral Sclerosis

(2014) Priyanka Patel et al.   MOLECULAR THERAPY

Nanobodies: Natural Single-Domain Antibodies

(2013) Serge Muyldermans   Annual Review of Biochemistry

Nanoparticle- and Liposome-carried Drugs: New Strategies for Active Targeting and Drug Delivery Across Blood-brain Barrier

(2013) Martha Pinzon-Daza et al.   CURRENT DRUG METABOLISM

Clinical implications of recent breakthroughs in amyotrophic lateral sclerosis

(2013) Philip Van Damme et al.   CURRENT OPINION IN NEUROLOGY

Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis

(2013) Hongyan Wang et al.   HUMAN MOLECULAR GENETICS

Minimally invasive transplantation of iPSC-derived ALDHhiSSCloVLA4+ neural stem cells effectively improves the phenotype of an amyotrophic lateral sclerosis model

(2013) Monica Nizzardo et al.   HUMAN MOLECULAR GENETICS

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

Transporters of the blood–brain and blood–CSF interfaces in development and in the adult

(2013) Norman R. Saunders et al.   MOLECULAR ASPECTS OF MEDICINE

Development, maintenance and disruption of the blood-brain barrier

(2013) Birgit Obermeier et al.   NATURE MEDICINE

State of play in amyotrophic lateral sclerosis genetics

(2013) Alan E Renton et al.   NATURE NEUROSCIENCE

The changing scene of amyotrophic lateral sclerosis

(2013) Wim Robberecht et al.   NATURE REVIEWS NEUROSCIENCE

Frequency of C9orf72 repeat expansions in amyotrophic lateral sclerosis: a Belgian cohort study

(2013) Sarah Debray et al.   NEUROBIOLOGY OF AGING

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

(2013) Christopher J. Donnelly et al.   NEURON

The role of heat shock proteins in Amyotrophic Lateral Sclerosis: The therapeutic potential of Arimoclomol

(2013) Bernadett Kalmar et al.   PHARMACOLOGY & THERAPEUTICS

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

(2013) C. Lagier-Tourenne et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Addressing Safety Liabilities of TfR Bispecific Antibodies That Cross the Blood-Brain Barrier

(2013) J. A. Couch et al.   Science Translational Medicine

Targeting RNA Foci in iPSC-Derived Motor Neurons from ALS Patients with a C9ORF72 Repeat Expansion

(2013) D. Sareen et al.   Science Translational Medicine

The genetics and neuropathology of amyotrophic lateral sclerosis

(2012) Ammar Al-Chalabi et al.   ACTA NEUROPATHOLOGICA

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(2012) Laura Canzi et al.   MOLECULAR MEDICINE

Autologous bone marrow-derived stem cells in amyotrophic lateral sclerosis: A pilot study

(2012) Sudesh Prabhakar et al.   NEUROLOGY INDIA

Intracerebroventricular Injection of Encapsulated Human Mesenchymal Cells Producing Glucagon-Like Peptide 1 Prolongs Survival in a Mouse Model of ALS

(2012) Sarah Knippenberg et al.   PLoS One

Stem cell technology for neurodegenerative diseases

(2011) J. Simon Lunn et al.   ANNALS OF NEUROLOGY

Research advances in gene therapy approaches for the treatment of amyotrophic lateral sclerosis

(2011) Monica Nizzardo et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

A C9orf72 promoter repeat expansion in a Flanders-Belgian cohort with disorders of the frontotemporal lobar degeneration-amyotrophic lateral sclerosis spectrum: a gene identification study

(2011) Ilse Gijselinck et al.   LANCET NEUROLOGY

Repeated courses of granulocyte colony-stimulating factor in amyotrophic lateral sclerosis: Clinical and biological results from a prospective multicenter study

(2011) Adriano Chiò et al.   MUSCLE & NERVE

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

(2011) Yimin Hua et al.   NATURE

The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis

(2011) Merit Cudkowicz et al.   NATURE MEDICINE

Gene therapy for amyotrophic lateral sclerosis

(2011) Thais Federici et al.   NEUROBIOLOGY OF DISEASE

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

(2011) Mariely DeJesus-Hernandez et al.   NEURON

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

(2011) Alan E. Renton et al.   NEURON

Intracerebroventricular Administration of Human Umbilical Cord Blood Cells Delays Disease Progression in Two Murine Models of Motor Neuron Degeneration

(2011) P. Bigini et al.   REJUVENATION RESEARCH

Safety and Immunological Effects of Mesenchymal Stem Cell Transplantation in Patients With Multiple Sclerosis and Amyotrophic Lateral Sclerosis

(2010) Dimitrios Karussis et al.   ARCHIVES OF NEUROLOGY

The neurobiology of amyotrophic lateral sclerosis

(2010) André Bento-Abreu et al.   EUROPEAN JOURNAL OF NEUROSCIENCE

Recent advances in motor neuron disease

(2009) Philip Van Damme et al.   CURRENT OPINION IN NEUROLOGY

Multiple administrations of human marrow stromal cells through cerebrospinal fluid prolong survival in a transgenic mouse model of amyotrophic lateral sclerosis

(2009) Chen Zhang et al.   CYTOTHERAPY

Brain drug targeting: a computational approach for overcoming blood–brain barrier

(2009) Ahmad Reza Mehdipour et al.   DRUG DISCOVERY TODAY

Intrathecal transplantation of human neural stem cells overexpressing VEGF provide behavioral improvement, disease onset delay and survival extension in transgenic ALS mice

(2009) D H Hwang et al.   GENE THERAPY

Chimerization of astroglial population in the lumbar spinal cord after mesenchymal stem cell transplantation prolongs survival in a rat model of amyotrophic lateral sclerosis

(2009) Cédric Boucherie et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Approaches to transport therapeutic drugs across the blood–brain barrier to treat brain diseases

(2009) Reinhard Gabathuler   NEUROBIOLOGY OF DISEASE

Dose-dependent efficacy of ALS-human mesenchymal stem cells transplantation into cisterna magna in SOD1-G93A ALS mice

(2009) Heejaung Kim et al.   NEUROSCIENCE LETTERS

Therapy development for ALS: Lessons learned and path forward

(2008) Veena Lanka et al.   Amyotrophic Lateral Sclerosis

Gene-Targeted Therapies for the Central Nervous System

(2008) Timothy M. Miller et al.   ARCHIVES OF NEUROLOGY

Novel Role for Vascular Endothelial Growth Factor (VEGF) Receptor-1 and Its Ligand VEGF-B in Motor Neuron Degeneration

(2008) K. Poesen et al.   JOURNAL OF NEUROSCIENCE

Therapeutic benefits of intrathecal protein therapy in a mouse model of amyotrophic lateral sclerosis

(2008) Yasuyuki Ohta et al.   JOURNAL OF NEUROSCIENCE RESEARCH