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References from From optical control to translational readiness: an evidence-tiered framework for optogenetics in neuromuscular and neurological disorders. Local targets link to admitted publications; unresolved targets remain external evidence.
Millisecond-timescale, genetically targeted optical control of neural activity
10.1038/nn1525 · 2005 · External reference
Optogenetics: 10 years of microbial opsins in neuroscience
10.1038/nn.4091 · 2015 · External reference
Integration of optogenetics with complementary methodologies in systems neuroscience
10.1038/nrn.2017.15 · 2017 · External reference
Optogenetics for light control of biological systems
10.1038/s43586-022-00136-4 · 2022 · External reference
All-optical electrophysiology for high-throughput functional characterization of a human iPSC-Derived motor neuron model of ALS
10.1016/j.stemcr.2018.04.020 · 2018 · External reference
RNA binding antagonizes neurotoxic phase transitions of TDP-43
10.1016/j.neuron.2019.01.048 · 2019 · External reference
Microfluidic device for the formation of optically excitable, three-dimensional, compartmentalized motor units
10.1126/sciadv.1501429 · 2016 · External reference
Memory retrieval by activating engram cells in mouse models of early Alzheimer's disease
10.1038/nature17172 · 2016 · External reference
Partial recovery of visual function in a blind patient after optogenetic therapy
10.1038/s41591-021-01351-4 · 2021 · External reference
Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?
10.1038/nrn2670 · 2009 · External reference
Challenges and opportunities in spinal muscular atrophy therapeutics
10.1016/s1474-4422(23)00419-2 · 2024 · External reference
Spinal muscular atrophy
10.1038/s41572-022-00380-8 · 2022 · External reference
Amyotrophic lateral sclerosis
10.1056/nejm200105313442207 · 2001 · External reference
Duchenne muscular dystrophy
10.1038/s41572-021-00248-3 · 2021 · External reference
Myasthenia gravis
10.1038/s41572-019-0079-y · 2019 · External reference
Regulation of parkinsonian motor behaviours by optogenetic control of basal ganglia circuitry
10.1038/nature09159 · 2010 · External reference
Gamma frequency entrainment attenuates amyloid load and modifies microglia
10.1038/nature20587 · 2016 · External reference
M2 cortex-dorsolateral striatum stimulation reverses motor symptoms and synaptic deficits in Huntington's disease
10.7554/elife.57017 · 2020 · External reference
Optogenetic neuronal stimulation promotes functional recovery after stroke
10.1073/pnas.1404109111 · 2014 · External reference
Medial septal GABAergic neurons reduce seizure duration upon optogenetic closed-loop stimulation
10.1093/brain/awab042 · 2021 · External reference
Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humans
10.1038/s41593-025-02097-9 · 2025 · External reference
Material and design strategies for chronically-implantable neural probes
10.1038/s41427-025-00613-8 · 2025 · External reference
Adaptive biomimicry: design of neural interfaces with enhanced biointegration
10.1016/j.copbio.2021.10.004 · 2021 · External reference
Technological challenges in the development of optogenetic closed-loop therapy approaches in epilepsy and related network disorders of the brain
10.3390/mi12010038 · 2020 · External reference
Viral vectors in gene therapy: where do we stand in 2023?
10.3390/v15030698 · 2023 · External reference
Towards translational optogenetics
10.1038/s41551-021-00829-3 · 2023 · External reference
Optogenetics 2.0: challenges and solutions towards a quantitative probing of neural circuits
10.1515/hsz-2023-0194 · 2024 · External reference
Channelrhodopsin-2, a directly light-gated cation-selective membrane channel
10.1073/pnas.1936192100 · 2003 · External reference
Light activation of channelrhodopsin-2 in excitable cells of Caenorhabditis elegans triggers rapid behavioral responses
10.1016/j.cub.2005.11.032 · 2005 · External reference
A user's guide to channelrhodopsin variants: features, limitations and future developments
10.1113/expphysiol.2009.051961 · 2011 · External reference
Ultrafast optogenetic control
10.1038/nn.2495 · 2010 · External reference
Bi-stable neural state switches
10.1038/nn.2247 · 2009 · External reference
Rhodopsin optogenetic toolbox v2.0 for light-sensitive excitation and inhibition in Caenorhabditis elegans
10.1371/journal.pone.0191802 · 2018 · External reference
An ultra-sensitive step-function opsin for minimally invasive optogenetic stimulation in mice and macaques
10.1016/j.neuron.2020.03.032 · 2020 · External reference
Independent optical excitation of distinct neural populations
10.1038/nmeth.2836 · 2014 · External reference
Practical considerations in an era of multicolor optogenetics
10.3389/fncel.2023.1160245 · 2023 · External reference
A bistable inhibitory optoGPCR for multiplexed optogenetic control of neural circuits
10.1038/s41592-024-02285-8 · 2024 · External reference
A single-vector intersectional AAV strategy for interrogating cellular diversity and brain function
10.1038/s41593-024-01659-7 · 2024 · External reference
Enhancer AAV toolbox for accessing and perturbing striatal cell types and circuits
2025 · External reference
Common AAV gene therapy vectors show nonselective transduction of ex vivo human brain tissue
10.1016/j.omtm.2025.101494 · 2025 · External reference
Adeno-associated virus (AAV) as a vector for gene therapy
10.1007/s40259-017-0234-5 · 2017 · External reference
Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration
10.1016/j.neuron.2006.02.026 · 2006 · External reference
Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration
10.1038/nn.2117 · 2008 · External reference
Optogenetics and targeted gene therapy for retinal diseases: unravelling the fundamentals, applications, and future perspectives
10.3390/jcm13144224 · 2024 · External reference
Optogenetics for understanding and treating brain injury: advances in the field and future prospects
10.3390/ijms23031800 · 2022 · External reference
Optogenetic modulation for the treatment of traumatic brain injury
10.1089/scd.2019.0187 · 2020 · External reference
10.1016/j.csbj.2022.06.061 · 2022 · Admitted local publication
Toward the optical cochlear implant
10.1101/cshperspect.a033225 · 2019 · External reference
Towards the optical cochlear implant: optogenetic approaches for hearing restoration
10.15252/emmm.201911618 · 2020 · External reference
Toward optogenetic hearing restoration
10.1146/annurev-neuro-070623-103247 · 2024 · External reference
Wireless and battery-free technologies for neuroengineering
10.1038/s41551-021-00683-3 · 2023 · External reference
Nano-optogenetics for disease therapies
10.1021/acsnano.4c00698 · 2024 · External reference
Microphysiological 3D model of amyotrophic lateral sclerosis (ALS) from human iPS-derived muscle cells and optogenetic motor neurons
10.1126/sciadv.aat5847 · 2018 · External reference
iPSC-derived functional human neuromuscular junctions model the pathophysiology of neuromuscular diseases
10.1172/jci.insight.124299 · 2019 · External reference
Optogenetic modeling of human neuromuscular circuits in Duchenne muscular dystrophy with CRISPR and pharmacological corrections
10.1126/sciadv.abi8787 · 2021 · External reference
Efficient generation of a self-organizing neuromuscular junction model from human pluripotent stem cells
10.1038/s41467-023-43781-3 · 2023 · External reference
Chronic optogenetic induction of stress granules is cytotoxic and reveals the evolution of ALS-FTD pathology
10.7554/elife.39578 · 2019 · External reference
TOR signaling regulates liquid phase separation of the SMN complex governing snRNP biogenesis
10.1016/j.celrep.2021.109277 · 2021 · External reference
Unraveling ChR2-driven stochastic Ca2+ dynamics in astrocytes: a call for new interventional paradigms
10.1371/journal.pcbi.1008648 · 2021 · External reference
Amyotrophic lateral sclerosis
10.1056/nejmra1603471 · 2017 · External reference
SMN in spinal muscular atrophy and snRNP biogenesis
10.1002/wrna.76 · 2011 · External reference
An optimized optogenetic clustering tool for probing protein interaction and function
10.1038/ncomms5925 · 2014 · External reference
Optogenetic TDP-43 nucleation induces persistent insoluble species and progressive motor dysfunction in vivo
10.1016/j.nbd.2020.105078 · 2020 · External reference
Spatiotemporal control of intracellular phase transitions using light-activated optoDroplets
10.1016/j.cell.2016.11.054 · 2017 · External reference
Phase separation by low complexity domains promotes stress granule assembly and drives pathological fibrillization
10.1016/j.cell.2015.09.015 · 2015 · External reference
Principles and properties of stress granules
10.1016/j.tcb.2016.05.004 · 2016 · External reference
ALS mutant FUS disrupts nuclear localization and sequesters wild-type FUS within cytoplasmic stress granules
10.1093/hmg/ddt117 · 2013 · External reference
ALS/FTD mutation-induced phase transition of FUS liquid droplets and reversible hydrogels into irreversible hydrogels impairs RNP granule function
10.1016/j.neuron.2015.10.030 · 2015 · External reference
A liquid-to-solid phase transition of the ALS protein FUS accelerated by disease mutation
10.1016/j.cell.2015.07.047 · 2015 · External reference
Loss of dynamic RNA interaction and aberrant phase separation induced by two distinct types of ALS/FTD-Linked FUS mutations
10.1016/j.molcel.2019.09.022 · 2020 · External reference
Aberrant phase separation of FUS leads to lysosome sequestering and acidification
10.3389/fcell.2021.716919 · 2021 · External reference
Puralpha: a multifunctional single-stranded DNA- and RNA-binding protein
10.1093/nar/28.17.3197 · 2000 · External reference
Expanded GGGGCC repeat RNA associated with amyotrophic lateral sclerosis and frontotemporal dementia causes neurodegeneration
10.1073/pnas.1219643110 · 2013 · External reference
Optogenetic control of GGGGCC repeat-containing RNA phase transition
10.1016/j.fmre.2022.09.001 · 2022 · External reference
C9orf72 poly-PR forms anisotropic condensates causative of nuclear TDP-43 pathology
10.1016/j.isci.2024.110937 · 2024 · External reference
Serotonergic dysfunction impairs locomotor coordination in spinal muscular atrophy
10.1093/brain/awad221 · 2023 · External reference
Electrophysiological properties of motor neurons in a mouse model of severe spinal muscular atrophy: in vitro versus in vivo development
10.1371/journal.pone.0011696 · 2010 · External reference
Synaptic disruption and CREB-regulated transcription are restored by K(+) channel blockers in ALS
10.15252/emmm.202013131 · 2021 · External reference
Repeat-associated non-AUG translation in C9orf72-ALS/FTD is driven by neuronal excitation and stress
10.15252/emmm.201809423 · 2019 · External reference
Cytosolic calcium regulates cytoplasmic accumulation of TDP-43 through Calpain-A and importin α3
10.7554/elife.60132 · 2020 · External reference
Brain-derived neurotrophic factor/tropomyosin receptor kinase B signaling in spinal muscular atrophy and amyotrophic lateral sclerosis
10.1016/j.nbd.2023.106377 · 2024 · External reference
Impaired muscle mitochondrial biogenesis and myogenesis in spinal muscular atrophy
10.1001/jamaneurol.2015.0178 · 2015 · External reference
SMN promotes mitochondrial metabolic maturation during myogenesis by regulating the MYOD-miRNA axis
10.26508/lsa.202201457 · 2023 · External reference
A customizable microfluidic platform for medium-throughput modeling of neuromuscular circuits
10.1016/j.biomaterials.2019.119537 · 2019 · External reference
On-chip 3D neuromuscular model for drug screening and precision medicine in neuromuscular disease
10.1038/s41596-019-0248-1 · 2020 · External reference
3D compartmentalised human pluripotent stem cell-derived neuromuscular Co-cultures
10.21769/bioprotoc.4624 · 2023 · External reference
A scalable human iPSC-based neuromuscular disease model on suspended biobased elastomer nanofiber scaffolds
10.1088/1758-5090/acf39e · 2023 · External reference
Spinal muscular atrophy: from approved therapies to future therapeutic targets for personalized medicine
2021 · External reference
Beyond motor neurons in spinal muscular atrophy: a focus on neuromuscular junction
10.3390/ijms25137311 · 2024 · External reference
Skeletal muscle in amyotrophic lateral sclerosis
10.1093/brain/awad202 · 2023 · External reference
The role of skeletal muscle in amyotrophic lateral sclerosis: a ‘dying‐back’ or ‘dying‐forward’ phenomenon?
10.1113/jp278835 · 2019 · External reference
A muscle fatigue-like contractile decline was recapitulated using skeletal myotubes from Duchenne muscular dystrophy patient-derived iPSCs
2021 · External reference
International consensus guidance for management of Myasthenia Gravis: 2020 update
10.1212/wnl.0000000000011124 · 2021 · External reference
A clinical perspective on muscle specific kinase antibody positive myasthenia gravis
10.3389/fimmu.2024.1502480 · 2024 · External reference
An optogenetic cell therapy to restore control of target muscles in an aggressive mouse model of amyotrophic lateral sclerosis
10.7554/elife.88250 · 2024 · External reference
Single-dose gene-replacement therapy for spinal muscular atrophy
10.1056/nejmoa1706198 · 2017 · External reference
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
10.1016/s1474-4422(21)00367-7 · 2022 · External reference
Optical deconstruction of parkinsonian neural circuitry
10.1126/science.1167093 · 2009 · External reference
Optogenetic gamma stimulation rescues memory impairments in an Alzheimer's disease mouse model
10.1038/s41467-019-13260-9 · 2019 · External reference
On-demand optogenetic control of spontaneous seizures in temporal lobe epilepsy
10.1038/ncomms2376 · 2013 · External reference
Closed-loop optogenetic control of thalamus as a tool for interrupting seizures after cortical injury
10.1038/nn.3269 · 2013 · External reference
Nine-year prospective efficacy and safety of brain-responsive neurostimulation for focal epilepsy
10.1212/wnl.0000000000010154 · 2020 · External reference
Neurostimulation treatments for epilepsy: deep brain stimulation, responsive neurostimulation and vagus nerve stimulation
10.1016/j.neurot.2023.e00308 · 2024 · External reference
Neurostimulation for stroke rehabilitation
10.3389/fnins.2021.649459 · 2021 · External reference
Ageing and Parkinson's disease: substantia nigra regional selectivity
10.1093/brain/114.5.2283 · 1991 · External reference
Anatomy, pigmentation, ventral and dorsal subpopulations of the substantia nigra, and differential cell death in Parkinson's disease
10.1136/jnnp.54.5.388 · 1991 · External reference
Oxidative stress in the aging substantia nigra and the etiology of Parkinson's disease
10.1111/acel.13031 · 2019 · External reference
Therapeutic deep brain stimulation disrupts movement-related subthalamic nucleus activity in parkinsonian mice
10.7554/elife.75253 · 2022 · External reference
Deep brain stimulation-guided optogenetic rescue of parkinsonian symptoms
10.1038/s41467-020-16046-6 · 2020 · External reference
Integrating artificial intelligence and optogenetics for Parkinson's disease diagnosis and therapeutics in male mice
10.1038/s41467-025-63025-w · 2025 · External reference
Optogenetics enables functional analysis of human embryonic stem cell-derived grafts in a Parkinson's disease model
10.1038/nbt.3124 · 2015 · External reference
The amyloid hypothesis of Alzheimer's disease at 25 years
10.15252/emmm.201606210 · 2016 · External reference
Alzheimer disease: an update on pathobiology and treatment strategies
10.1016/j.cell.2019.09.001 · 2019 · External reference
Neuronal hyperexcitability in Alzheimer's disease: what are the drivers behind this aberrant phenotype?
10.1038/s41398-022-02024-7 · 2022 · External reference
Multi-sensory Gamma stimulation ameliorates alzheimer's-associated pathology and improves cognition
10.1016/j.cell.2019.02.014 · 2019 · External reference
Driving GABAergic neurons optogenetically improves learning, reduces amyloid load and enhances autophagy in a mouse model of Alzheimer's disease
10.1016/j.bbrc.2020.03.004 · 2020 · External reference
Sleep restoration by optogenetic targeting of GABAergic neurons reprograms microglia and ameliorates pathological phenotypes in an Alzheimer's disease model
10.1186/s13024-023-00682-9 · 2023 · External reference
Basal forebrain cholinergic circuits and signaling in cognition and cognitive decline
10.1016/j.neuron.2016.09.006 · 2016 · External reference
Exploring the pathogenesis of alzheimer disease in basal forebrain cholinergic neurons: converging insights from alternative hypotheses
10.3389/fnins.2019.00446 · 2019 · External reference
Chemical genetic activation of the cholinergic basal forebrain hippocampal circuit rescues memory loss in Alzheimer's disease
10.1186/s13195-022-00994-w · 2022 · External reference
Optogenetics in Alzheimer's Disease: focus on astrocytes
10.3390/antiox12101856 · 2023 · External reference
Complement and microglia mediate early synapse loss in Alzheimer mouse models
10.1126/science.aad8373 · 2016 · External reference
Clearance of β-amyloid and synapses by the optogenetic depolarization of microglia is complement selective
10.1016/j.neuron.2023.12.003 · 2024 · External reference
Homeostatic microglia initially seed and activated microglia later reshape amyloid plaques in Alzheimer's disease
10.1038/s41467-024-54779-w · 2024 · External reference
Huntington disease
10.1038/nrdp.2015.5 · 2015 · External reference
Differential impact of optogenetic stimulation of direct and indirect pathways from dorsolateral and dorsomedial striatum on motor symptoms in Huntington's disease mice
10.1016/j.expneurol.2024.114991 · 2025 · External reference
Encouraging an excitable brain state: mechanisms of brain repair in stroke
10.1038/s41583-020-00396-7 · 2021 · External reference
Rethinking remapping: circuit mechanisms of recovery after stroke
10.1523/jneurosci.1425-23.2023 · 2023 · External reference
Optogenetic stimulation enhanced neuronal plasticities in motor recovery after ischemic stroke
10.1155/2019/5271573 · 2019 · External reference
Brain-wide neural dynamics of poststroke recovery induced by optogenetic stimulation
10.1126/sciadv.abd9465 · 2021 · External reference
Homotopic contralesional excitation suppresses spontaneous circuit repair and global network reconnections following ischemic stroke
10.7554/elife.68852 · 2022 · External reference
Ischemic stroke rehabilitation through optogenetic modulation of parvalbumin neurons in the contralateral motor cortex
10.1016/j.expneurol.2022.114289 · 2023 · External reference
Neuron type-specific optogenetic stimulation for differential stroke recovery in chronic capsular infarct
10.1038/s12276-024-01253-8 · 2024 · External reference
Gamma frequency activation of inhibitory neurons in the acute phase after stroke attenuates vascular and behavioral dysfunction
10.1016/j.celrep.2021.108696 · 2021 · External reference
40-Hz optogenetic stimulation rescues functional synaptic plasticity after stroke
10.1016/j.celrep.2023.113475 · 2023 · External reference
Optogenetic calcium modulation in astrocytes enhances post-stroke recovery in chronic capsular infarct
10.1126/sciadv.adn7577 · 2025 · External reference
Optogenetic control of epileptiform activity
10.1073/pnas.0901915106 · 2009 · External reference
In vivo evaluation of the dentate gate theory in epilepsy
10.1113/jp270056 · 2015 · External reference
Inhibitory or excitatory? Optogenetic interrogation of the functional roles of GABAergic interneurons in epileptogenesis
10.1186/s12929-017-0399-8 · 2017 · External reference
Graded optogenetic activation of the auditory pathway for hearing restoration
10.1016/j.brs.2023.01.1671 · 2023 · External reference
Spatiotemporal, optogenetic control of gene expression in organoids
10.1038/s41592-023-01986-w · 2023 · External reference
Revealing single-neuron and network-activity interaction by combining high-density microelectrode array and optogenetics
10.1038/s41467-024-53505-w · 2024 · External reference
Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial
10.1016/j.eclinm.2025.103430 · 2025 · External reference
Improved optogenetic modification of spiral ganglion neurons for future optical cochlear implants
10.7150/thno.104474 · 2025 · External reference
Advances and challenges in gene therapy for neurodegenerative diseases: a systematic review
10.3390/ijms252312485 · 2024 · External reference
Chronically implantable μLED arrays for optogenetic cortical surface stimulation in mice
10.1038/s41467-025-68191-5 · 2026 · External reference
Transcranial optogenetic brain modulator for precise bimodal neuromodulation in multiple brain regions
10.1038/s41467-024-54759-0 · 2024 · External reference
Fully bioresorbable hybrid opto-electronic neural implant system for simultaneous electrophysiological recording and optogenetic stimulation
10.1038/s41467-024-45803-0 · 2024 · External reference
A wireless optogenetic stimulation system for long-term function evaluation of mice forelimb with sub-nerve resolution
10.1038/s41467-025-63746-y · 2025 · External reference
Deep brain stimulation for parkinson's Disease-A narrative review
10.3390/biomedicines13102430 · 2025 · External reference
A large-scale optogenetic neurophysiology platform for improving accessibility in non-human primate behavioral experiments
10.1038/s41467-026-69448-3 · 2026 · External reference
On brain stimulation in epilepsy
10.1093/brain/awae385 · 2025 · External reference
Model-based optogenetic stimulation to regulate beta oscillations in Parkinsonian neural networks
10.1007/s11571-021-09729-3 · 2022 · External reference
Dynamic analysis of beta oscillations in Parkinsonian neural networks with the pedunculopontine nucleus under optogenetic control
10.1007/s10409-024-23586-x · 2024 · External reference
GABAergic interneurons in seizures: investigating causality with optogenetics
10.1177/1073858418805002 · 2019 · External reference
Optogenetic intervention of seizures improves spatial memory in a mouse model of chronic temporal lobe epilepsy
10.1111/epi.16445 · 2020 · External reference
Depolarized GABAergic signaling in subicular microcircuits mediates generalized seizure in temporal Lobe Epilepsy
10.1016/j.neuron.2017.06.004 · 2017 · External reference
Adult-born neurons in critical period maintain hippocampal seizures via local aberrant excitatory circuits
10.1038/s41392-023-01433-4 · 2023 · External reference
Optogenetic stimulation of the dorsal striatum bidirectionally controls seizures
10.1073/pnas.2419178122 · 2025 · External reference
Minimally-invasive manipulation of spared and hypoactive interneurons reduces CA1 synchronization and nonspatial behavior alterations in epilepsy models
10.1016/j.nbd.2025.107155 · 2025 · External reference
Generalization—A key challenge for responsible AI in patient-facing clinical applications
10.1038/s41746-024-01127-3 · 2024 · External reference
A scoping review of robustness concepts for machine learning in healthcare
10.1038/s41746-024-01420-1 · 2025 · External reference
Recalibration of neuromodulation parameters in neural implants with adaptive Bayesian optimization
10.1088/1741-2552/acc975 · 2023 · External reference
Designing and validating a robust adaptive neuromodulation algorithm for closed-loop control of brain states
10.1088/1741-2552/ac7005 · 2022 · External reference
Unresolved reference
2025 · External reference
Optogenetics stimulates nerve reorganization in the contralesional anterolateral primary motor cortex in a mouse model of ischemic stroke
10.4103/1673-5374.330615 · 2022 · External reference
Neurophotonic methods in approach to in vivo animal epileptic models: advantages and limitations
10.1111/epi.17870 · 2024 · External reference
The effect of the optogenetic stimulation of astrocytes on neural network activity in an in vitro model of Alzheimer's disease
10.3390/ijms252212237 · 2024 · External reference
D1R- and D2R-Medium-Sized spiny neurons diversity: insights into striatal vulnerability to Huntington's disease mutation
10.3389/fncel.2021.628010 · 2021 · External reference
Striatal direct and indirect pathway output structures are differentially altered in mouse models of Huntington's disease
10.1523/jneurosci.0434-18.2018 · 2018 · External reference
Dysregulation of corticostriatal connectivity in Huntington's disease: a role for dopamine modulation
10.3233/jhd-160221 · 2016 · External reference
Corticostriatal network dysfunction in Huntington's disease: deficits in neural processing, glutamate transport, and ascorbate release
10.1111/cns.12828 · 2018 · External reference
Disrupted striatal neuron inputs and outputs in Huntington's disease
10.1111/cns.12844 · 2018 · External reference