Challenges for Therapeutic Applications of Opsin-Based Optogenetic Tools in Humans

Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes?

(2020) Patrizia Tornabene et al.   HUMAN GENE THERAPY

Enhancer-Driven Gene Expression (EDGE) enables the generation of cell type specific tools for the analysis of neural circuits

(2020) Stefan Blankvoort et al.   NEUROSCIENCE RESEARCH

Anisotropic light scattering from myelinated axons in the spinal cord

(2020) Damon DePaoli et al.   Neurophotonics

Theranostic Strategy of Focused Ultrasound Induced Blood-Brain Barrier Opening for CNS Disease Treatment

(2019) Ko-Ting Chen et al.   Frontiers in Pharmacology

Restoration of high-sensitivity and adapting vision with a cone opsin

(2019) Michael H. Berry et al.   Nature Communications

Enhanced Social Dominance and Altered Neuronal Excitability in the Prefrontal Cortex of Male KCC2b Mutant Mice

(2019) Allison M. J. Anacker et al.   Autism Research

Safe and sustained expression of human iduronidase after adeno-associated virus 9 intrathecal administration in infant rhesus monkeys

(2019) Juliette Hordeaux et al.   HUMAN GENE THERAPY

Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality

(2019) Eloise Hudry et al.   NEURON

The awesome power of optogenetics in hearing research

(2019) Joseph DiGuiseppi et al.   NEUROSCIENCE LETTERS

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

(2019) Patrizia Tornabene et al.   Science Translational Medicine

In Vitro Gene Delivery in Retinal Pigment Epithelium Cells by Plasmid DNA-Wrapped Gold Nanoparticles

(2019) Trigueros et al.   Genes

Next-generation AAV vectors - don't judge a virus (only) by its cover

(2019) Claire Domenger et al.   HUMAN MOLECULAR GENETICS

Publisher Correction: Systemic AAV vectors for widespread and targeted gene delivery in rodents

(2019) Rosemary C. Challis et al.   Nature Protocols

Cortical layer–specific critical dynamics triggering perception

(2019) James H. Marshel et al.   SCIENCE

Optogenetics in the Era of Cerebral Organoids

(2019) Zahra Shiri et al.   TRENDS IN BIOTECHNOLOGY

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation

(2019) Aaron S. Meadows et al.   Molecular Therapy-Methods & Clinical Development

AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice

(2019) William A. Liguore et al.   MOLECULAR THERAPY

Optogenetic and chemogenetic therapies for epilepsy

(2019) Matthew C. Walker et al.   NEUROPHARMACOLOGY

Development of Efficient AAV2/DJ-Based Viral Vectors to Selectively Downregulate the Expression of Neuronal or Astrocytic Target Proteins in the Rat Central Nervous System

(2019) Charlotte Jollé et al.   Frontiers in Molecular Neuroscience

Viral-Mediated Optogenetic Stimulation of Peripheral Motor Nerves in Non-human Primates

(2019) Jordan J. Williams et al.   Frontiers in Neuroscience

Machine learning-guided channelrhodopsin engineering enables minimally invasive optogenetics

(2019) Claire N. Bedbrook et al.   NATURE METHODS

Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids

(2019) Qin Huang et al.   PLoS One

A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo

(2019) Guocai Zhong et al.   NATURE BIOTECHNOLOGY

Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS

(2019) Mariana Bravo-Hernandez et al.   NATURE MEDICINE

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

(2019) Natalie F. Nidetz et al.   PHARMACOLOGY & THERAPEUTICS

Visceral pain – Novel approaches for optogenetic control of spinal afferents

(2018) Nick J. Spencer et al.   BRAIN RESEARCH

Marked Diversity of Unique Cortical Enhancers Enables Neuron-Specific Tools by Enhancer-Driven Gene Expression

(2018) Stefan Blankvoort et al.   CURRENT BIOLOGY

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

A bright future? Optogenetics in the periphery for pain research and therapy

(2018) Aaron D. Mickle et al.   PAIN

Innovative Optogenetic Strategies for Vision Restoration

(2018) Cameron K. Baker et al.   Frontiers in Cellular Neuroscience

Optogenetic Peripheral Nerve Immunogenicity

(2018) Benjamin E. Maimon et al.   Scientific Reports

A wireless closed-loop system for optogenetic peripheral neuromodulation

(2018) Aaron D. Mickle et al.   NATURE

AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials

(2017) Nathan Hardcastle et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Nonhuman Primate Optogenetics: Recent Advances and Future Directions

(2017) Adriana Galvan et al.   JOURNAL OF NEUROSCIENCE

Site-Specific PEGylated Adeno-Associated Viruses with Increased Serum Stability and Reduced Immunogenicity

(2017) Tianzhuo Yao et al.   MOLECULES

Blocking microglial pannexin-1 channels alleviates morphine withdrawal in rodents

(2017) Nicole E Burma et al.   NATURE MEDICINE

Optogenetic control with a photocleavable protein, PhoCl

(2017) Wei Zhang et al.   NATURE METHODS

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Flexible Near-Field Wireless Optoelectronics as Subdermal Implants for Broad Applications in Optogenetics

(2017) Gunchul Shin et al.   NEURON

AAV-Mediated Anterograde Transsynaptic Tagging: Mapping Corticocollicular Input-Defined Neural Pathways for Defense Behaviors

(2017) Brian Zingg et al.   NEURON

Optogenetic Tools for Subcellular Applications in Neuroscience

(2017) Benjamin R. Rost et al.   NEURON

Cationic carbon quantum dots derived from alginate for gene delivery: One-step synthesis and cellular uptake

(2016) Jie Zhou et al.   Acta Biomaterialia

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

Epidural optogenetics for controlled analgesia

(2016) Robert P Bonin et al.   Molecular Pain

AAVR: A Multi-Serotype Receptor for AAV

(2016) Candace Summerford et al.   MOLECULAR THERAPY

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Stress Increases Ethanol Self-Administration via a Shift toward Excitatory GABA Signaling in the Ventral Tegmental Area

(2016) Alexey Ostroumov et al.   NEURON

A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons

(2016) D. Gowanlock R. Tervo et al.   NEURON

Probing pain pathways with light

(2016) Feng Wang et al.   NEUROSCIENCE

Interaction of vectors and parental viruses with the host genome

(2016) Irene Gil-Farina et al.   Current Opinion in Virology

In vivo tissue-tropism of adeno-associated viral vectors

(2016) Arun Srivastava   Current Opinion in Virology

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

(2016) Kyle Chamberlain et al.   Human Gene Therapy Methods

Optogenetic dissection of neural circuitry: from synaptic causalities to blue prints for novel treatments of behavioral diseases

(2015) Christian Lüscher et al.   CURRENT OPINION IN NEUROBIOLOGY

Strategies to circumvent humoral immunity to adeno-associated viral vectors

(2015) Longping V Tse et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Local and systemic responses following intravitreous injection of AAV2-encoded modified Volvox channelrhodopsin-1 in a genetically blind rat model

(2015) E Sugano et al.   GENE THERAPY

Optogenetics: 10 years of microbial opsins in neuroscience

(2015) Karl Deisseroth   NATURE NEUROSCIENCE

Natural light-gated anion channels: A family of microbial rhodopsins for advanced optogenetics

(2015) E. G. Govorunova et al.   SCIENCE

An improved chloride-conducting channelrhodopsin for light-induced inhibition of neuronal activity in vivo

(2015) Jonas Wietek et al.   Scientific Reports

Independent optical excitation of distinct neural populations

(2014) Nathan C Klapoetke et al.   NATURE METHODS

All-optical electrophysiology in mammalian neurons using engineered microbial rhodopsins

(2014) Daniel R Hochbaum et al.   NATURE METHODS

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Biology of adeno-associated viral vectors in the central nervous system

(2014) Giridhar Murlidharan et al.   Frontiers in Molecular Neuroscience

Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors

(2014) Charles N de Leeuw et al.   Molecular Therapy-Methods & Clinical Development

Morphine hyperalgesia gated through microglia-mediated disruption of neuronal Cl− homeostasis

(2013) Francesco Ferrini et al.   NATURE NEUROSCIENCE

ReaChR: a red-shifted variant of channelrhodopsin enables deep transcranial optogenetic excitation

(2013) John Y Lin et al.   NATURE NEUROSCIENCE

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

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

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Blockade of PDGFR-β activation eliminates morphine analgesic tolerance

(2012) Yan Wang et al.   NATURE MEDICINE

Optogenetic silencing strategies differ in their effects on inhibitory synaptic transmission

(2012) Joseph V Raimondo et al.   NATURE NEUROSCIENCE

Neocortical excitation/inhibition balance in information processing and social dysfunction

(2011) Ofer Yizhar et al.   NATURE

High-efficiency channelrhodopsins for fast neuronal stimulation at low light levels

(2011) A. Berndt et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Online object oriented Monte Carlo computational tool for the needs of biomedical optics

(2011) Alexander Doronin et al.   Biomedical Optics Express

Enhancing the clinical potential of AAV vectors by capsid engineering to evade pre-existing immunity

(2011) Melissa Bartel   Frontiers in Microbiology

Immune responses to adeno-associated virus type 2 encoding channelrhodopsin-2 in a genetically blind rat model for gene therapy

(2010) E Sugano et al.   GENE THERAPY

Ultrafast optogenetic control

(2010) Lisa A Gunaydin et al.   NATURE NEUROSCIENCE

Characterization of Engineered Channelrhodopsin Variants with Improved Properties and Kinetics

(2009) John Y. Lin et al.   BIOPHYSICAL JOURNAL

Resting-state functional connectivity in the human brain revealed with diffuse optical tomography

(2009) Brian R. White et al.   NEUROIMAGE

Red-shifted optogenetic excitation: a tool for fast neural control derived from Volvox carteri

(2008) Feng Zhang et al.   NATURE NEUROSCIENCE