| review article | Q7318358 |
| scholarly article | Q13442814 |
| P50 | author | Marisa Cappella | Q57571799 |
| P2093 | author name string | Maria Grazia Biferi | |
| Mathilde Cohen-Tannoudji | |||
| Chiara Ciotti | |||
| P2860 | cites work | Aggregation and Motor Neuron Toxicity of an ALS-Linked SOD1 Mutant Independent from Wild-Type SOD1 | Q22299419 |
| C9ORF72, implicated in amytrophic lateral sclerosis and frontotemporal dementia, regulates endosomal trafficking | Q24337586 | ||
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention | Q24562489 | ||
| RNA-guided human genome engineering via Cas9 | Q24598394 | ||
| Multiplex genome engineering using CRISPR/Cas systems | Q24609428 | ||
| Lentiviral vectors in gene therapy: their current status and future potential | Q24629462 | ||
| Expanded GGGGCC hexanucleotide repeat in noncoding region of C9ORF72 causes chromosome 9p-linked FTD and ALS | Q24633692 | ||
| A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD | Q24634583 | ||
| A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity | Q24669850 | ||
| State-of-the-art human gene therapy: part I. Gene delivery technologies | Q26825575 | ||
| State of play in amyotrophic lateral sclerosis genetics | Q26864816 | ||
| Amyotrophic lateral sclerosis and structural defects in Cu,Zn superoxide dismutase | Q27731976 | ||
| MicroRNAs: genomics, biogenesis, mechanism, and function | Q27861070 | ||
| Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis | Q28131672 | ||
| Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis | Q28131805 | ||
| Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study | Q28141072 | ||
| From Charcot to Lou Gehrig: deciphering selective motor neuron death in ALS | Q28206361 | ||
| Mutations in the FUS/TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis | Q28236796 | ||
| Mutations in FUS, an RNA processing protein, cause familial amyotrophic lateral sclerosis type 6 | Q28236805 | ||
| Mechanisms of gene silencing by double-stranded RNA | Q28282560 | ||
| Adeno-associated virus and lentivirus vectors: a refined toolkit for the central nervous system | Q38936840 | ||
| The Epidemiology of Neuromuscular Disorders: A Comprehensive Overview of the Literature | Q38960716 | ||
| Decoding ALS: from genes to mechanism. | Q39004348 | ||
| Genetic manipulation of specific neural circuits by use of a viral vector system | Q39069504 | ||
| Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases | Q39391538 | ||
| The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy | Q39662062 | ||
| A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model | Q40140649 | ||
| Neuroprotective effects of glial cell line-derived neurotrophic factor mediated by an adeno-associated virus vector in a transgenic animal model of amyotrophic lateral sclerosis. | Q40710699 | ||
| Isoform-specific antibodies reveal distinct subcellular localizations of C9orf72 in amyotrophic lateral sclerosis | Q40737530 | ||
| RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN | Q41275236 | ||
| Therapeutic strategies for spinal muscular atrophy: SMN and beyond. | Q41440571 | ||
| Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 | ||
| Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model | Q43628011 | ||
| Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy | Q44329621 | ||
| Intraspinal cord delivery of IGF-I mediated by adeno-associated virus 2 is neuroprotective in a rat model of familial ALS. | Q45387438 | ||
| Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice | Q45865368 | ||
| Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy | Q45873849 | ||
| Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis. | Q45881110 | ||
| Multifactorial Gene Therapy Enhancing the Glutamate Uptake System and Reducing Oxidative Stress Delays Symptom Onset and Prolongs Survival in the SOD1-G93A ALS Mouse Model | Q45881781 | ||
| P525L FUS mutation is consistently associated with a severe form of juvenile amyotrophic lateral sclerosis. | Q45894689 | ||
| Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy | Q46272554 | ||
| Treatment with edaravone, initiated at symptom onset, slows motor decline and decreases SOD1 deposition in ALS mice | Q46418017 | ||
| Motor Neuron Gene Therapy: Lessons from Spinal Muscular Atrophy for Amyotrophic Lateral Sclerosis | Q47152662 | ||
| In vivo genome editing improves motor function and extends survival in a mouse model of ALS. | Q47153099 | ||
| VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model | Q47427098 | ||
| Emerging Issues in AAV-Mediated In Vivo Gene Therapy | Q47554736 | ||
| Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. | Q48146867 | ||
| Nonviral siRNA delivery for gene silencing in neurodegenerative diseases. | Q48277611 | ||
| Retrogradely transported siRNA silences human mutant SOD1 in spinal cord motor neurons | Q48717565 | ||
| C9orf72 BAC Mouse Model with Motor Deficits and Neurodegenerative Features of ALS/FTD. | Q48794622 | ||
| Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy | Q50004193 | ||
| Viral vectors for therapy of neurologic diseases | Q38746238 | ||
| AAV-Mediated Gene Therapy for Research and Therapeutic Purposes | Q38766616 | ||
| Ex Vivo Gene Therapy Using Human Mesenchymal Stem Cells to Deliver Growth Factors in the Skeletal Muscle of a Familial ALS Rat Model | Q38816326 | ||
| The expanding biology of the C9orf72 nucleotide repeat expansion in neurodegenerative disease | Q38826893 | ||
| Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. | Q51659718 | ||
| Oligonucleotide therapeutics in neurodegenerative diseases. | Q51737325 | ||
| Amyotrophic Lateral Sclerosis. | Q53750443 | ||
| Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS. | Q55399094 | ||
| Amyotrophic lateral sclerosis | Q55877676 | ||
| Gene therapy for neurological disorders: progress and prospects | Q57165501 | ||
| CRISPR-mediated genome editing and human diseases | Q57253681 | ||
| Animal models of amyotrophic lateral sclerosis: A comparison of model validity | Q57455064 | ||
| Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques | Q58091980 | ||
| Non-viral Delivery of Nucleic Acids: Insight Into Mechanisms of Overcoming Intracellular Barriers | Q58728426 | ||
| C9orf72 deficiency promotes motor deficits of a C9ALS/FTD mouse model in a dose-dependent manner | Q64065925 | ||
| Targeting RNA-Mediated Toxicity in C9orf72 ALS and/or FTD by RNAi-Based Gene Therapy | Q64231298 | ||
| Artificial MicroRNAs Targeting C9orf72 Can Reduce Accumulation of Intra-nuclear Transcripts in ALS and FTD Patients | Q64250262 | ||
| C9orf72-mediated ALS and FTD: multiple pathways to disease. | Q64948055 | ||
| A controlled trial of riluzole in amyotrophic lateral sclerosis. ALS/Riluzole Study Group | Q72759362 | ||
| Background and gender effects on survival in the TgN(SOD1-G93A)1Gur mouse model of ALS | Q80384279 | ||
| Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models | Q90236625 | ||
| Amyotrophic Lateral Sclerosis Descriptive Epidemiology: The Origin of Geographic Difference | Q90851291 | ||
| Genetics of amyotrophic lateral sclerosis: A review | Q92359583 | ||
| Rats expressing human cytosolic copper-zinc superoxide dismutase transgenes with amyotrophic lateral sclerosis: associated mutations develop motor neuron disease | Q94029459 | ||
| The special Sm core structure of the U7 snRNP: far-reaching significance of a small nuclear ribonucleoprotein | Q28291688 | ||
| Motor neurons in Cu/Zn superoxide dismutase-deficient mice develop normally but exhibit enhanced cell death after axonal injury | Q28511628 | ||
| Motor neuron degeneration in mice that express a human Cu,Zn superoxide dismutase mutation | Q29547561 | ||
| TDP-43 is a component of ubiquitin-positive tau-negative inclusions in frontotemporal lobar degeneration and amyotrophic lateral sclerosis | Q29615597 | ||
| Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is trafficked in a highly motile Rab7-positive compartment | Q30573520 | ||
| Rabies virus envelope glycoprotein targets lentiviral vectors to the axonal retrograde pathway in motor neurons | Q30580046 | ||
| Adeno-associated virus-delivered artificial microRNA extends survival and delays paralysis in an amyotrophic lateral sclerosis mouse model | Q30843477 | ||
| Glial promoter selectivity following AAV-delivery to the immature brain | Q31120301 | ||
| Enhancement of SMN2 exon 7 inclusion by antisense oligonucleotides targeting the exon | Q33278100 | ||
| Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides | Q33635469 | ||
| Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons | Q33713769 | ||
| Amyotrophic lateral sclerosis: improving care with a multidisciplinary approach | Q33734415 | ||
| Incidence of amyotrophic lateral sclerosis in Europe | Q33774202 | ||
| Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion | Q33878156 | ||
| RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform | Q34020786 | ||
| Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy | Q34168139 | ||
| Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis | Q34224830 | ||
| A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse | Q34241874 | ||
| Unlocking the potential of the human genome with RNA interference | Q34348958 | ||
| Splicing of a critical exon of human Survival Motor Neuron is regulated by a unique silencer element located in the last intron | Q34353625 | ||
| AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice | Q34383015 | ||
| Immune responses to adeno-associated virus vectors | Q34428544 | ||
| Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy | Q34540158 | ||
| ALS Clinical Trials Review: 20 Years of Failure. Are We Any Closer to Registering a New Treatment? | Q34554820 | ||
| State-of-the-art human gene therapy: part II. Gene therapy strategies and clinical applications. | Q34664690 | ||
| Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone (MCI-186) in amyotrophic lateral sclerosis patients | Q34699979 | ||
| Self-complementary AAV vectors; advances and applications | Q34804660 | ||
| Antisense oligonucleotide therapy for neurodegenerative disease. | Q34830633 | ||
| Incidence and lifetime risk of motor neuron disease in the United Kingdom: a population-based study. | Q34976696 | ||
| Highly efficient retrograde gene transfer into motor neurons by a lentiviral vector pseudotyped with fusion glycoprotein | Q35006070 | ||
| Adeno-associated viruses undergo substantial evolution in primates during natural infections | Q35022838 | ||
| Viral vectors for gene delivery to the nervous system | Q35120134 | ||
| Progress and problems with the use of viral vectors for gene therapy | Q35120176 | ||
| Exome sequencing in amyotrophic lateral sclerosis identifies risk genes and pathways | Q35624305 | ||
| The AAV vector toolkit: poised at the clinical crossroads | Q35876224 | ||
| Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice | Q35894587 | ||
| Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo | Q35948485 | ||
| Patterns of Weakness, Classification of Motor Neuron Disease, and Clinical Diagnosis of Sporadic Amyotrophic Lateral Sclerosis | Q36238903 | ||
| C9orf72 BAC Transgenic Mice Display Typical Pathologic Features of ALS/FTD | Q36353773 | ||
| Lentiviral vectors for use in the central nervous system. | Q36364923 | ||
| Prognostic factors in ALS: A critical review | Q36447824 | ||
| Intraparenchymal spinal cord delivery of adeno-associated virus IGF-1 is protective in the SOD1G93A model of ALS | Q36491424 | ||
| Gene Therapy for ALS: progress and prospects | Q36522324 | ||
| Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates. | Q36543516 | ||
| Molecular biology of amyotrophic lateral sclerosis: insights from genetics | Q36573336 | ||
| Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy | Q36661721 | ||
| Human C9ORF72 Hexanucleotide Expansion Reproduces RNA Foci and Dipeptide Repeat Proteins but Not Neurodegeneration in BAC Transgenic Mice | Q36788842 | ||
| Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs | Q36880235 | ||
| Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience | Q36912314 | ||
| Immune responses to AAV in clinical trials | Q36990095 | ||
| An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study | Q37015657 | ||
| Projected increase in amyotrophic lateral sclerosis from 2015 to 2040 | Q37181233 | ||
| Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector | Q37251114 | ||
| Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity | Q37331730 | ||
| Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration | Q37340843 | ||
| Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS | Q37393810 | ||
| Lentiviral delivery of short hairpin RNAs | Q37430557 | ||
| Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis | Q37463967 | ||
| The evidence for altered RNA metabolism in amyotrophic lateral sclerosis (ALS). | Q37617743 | ||
| Additive amelioration of ALS by co-targeting independent pathogenic mechanisms | Q37620170 | ||
| AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS. | Q37622300 | ||
| Enhanced central nervous system transduction with lentiviral vectors pseudotyped with RVG/HIV-1gp41 chimeric envelope glycoproteins. | Q37643999 | ||
| Molecular pathways of motor neuron injury in amyotrophic lateral sclerosis | Q37952528 | ||
| Axonal transport deficits and neurodegenerative diseases | Q38077679 | ||
| Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies. | Q38177110 | ||
| Genetic causes of amyotrophic lateral sclerosis: new genetic analysis methodologies entailing new opportunities and challenges | Q38260359 | ||
| C9orf72 and RAB7L1 regulate vesicle trafficking in amyotrophic lateral sclerosis and frontotemporal dementia | Q38288577 | ||
| Gene and splicing therapies for neuromuscular diseases. | Q38471992 | ||
| Gene therapy for neurological disorders | Q38661071 | ||
| P275 | copyright license | Creative Commons Attribution | Q6905323 |
| P6216 | copyright status | copyrighted | Q50423863 |
| P433 | issue | 18 | |
| P921 | main subject | amyotrophic lateral sclerosis | Q206901 |
| gene therapy | Q213901 | ||
| P577 | publication date | 2019-09-06 | |
| P1433 | published in | International Journal of Molecular Sciences | Q3153277 |
| P1476 | title | Gene Therapy for ALS-A Perspective | |
| P478 | volume | 20 |
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