Toolkit Items

Emerging delivery vehicles (AAVs, LNPs, lentivirus, virus-like particles) and their translational implications are discussed.

Adeno-associated virus delivery is a viral gene delivery harness used to deploy the far-red light-induced split-Cre recombinase (FISC) system in vivo. In the cited study, AAV delivery enabled implementation of optogenetically controlled genome engineering in living systems.

Species D adenoviruses, such as human adenovirus type 10 (HAdV-D10), are promising candidates due to low seroprevalence in humans... support the advancement of HAdV-D10 as a next-generation platform for gene delivery and vaccine development.

Adenoviral infection is a viral delivery harness used in vitro to introduce the optogenetic actuators ChR2(H134R) and ArchT into primary cardiac fibroblasts. In the cited Methods in Molecular Biology protocol, it enables quick, robust, and consistent opsin expression and supports generation of light-responsive cardiac fibroblast preparations.

Recombinant AAV1/2 viral particles are a viral gene delivery harness used to express the LOV2-PAH1 optogenetic REST inhibitor in HEK293T cells, primary neurons, and mouse hippocampal neurons in vivo. In the cited study, these particles enabled efficient hippocampal neuronal transduction and subsequent functional testing of the delivered cargo.

Adenovirus is described here as a viral delivery harness used in optogenetic experiments to introduce genes encoding photosensitive proteins into specific neural regions. This delivery enables subsequent light-gated control of ion passage for neuronal activation or inhibition.

Viral vectors for ocular gene delivery are described as available tools that can support optogenetic strategies for vision restoration. The supplied evidence identifies their use context in the eye but does not specify vector type, cargo architecture, or cellular targets.

AAV-based viral vectors are adeno-associated virus delivery systems used to introduce optogenetic transgenes for expression in target cell types. In the cited therapeutic optogenetics context, they are presented as promising for human trials but still limited by barriers to general use.

Recombinant adeno-associated virus (rAAV) vectors are viral gene delivery vehicles used for in vitro and in vivo transgene delivery, including in the retina. In the cited retinal ganglion cell study, rAAV supported motif-mediated subcellular targeting of optogenetic tools to shape expression patterns.

AAV serotype (AAV2.2 versus engineered AAV2.NN)-were systematically optimized. Whole-retina-to-brain imaging was performed using fMOST on samples injected with the optimal condition (AAV2.2-double-1/1000).

AAV2.7m8 is a powerful viral vector for inner ear gene therapy

AAV5, oral administration of deschloroclozapine, and clozapine-N-oxide were also effective but with slightly less potency.

Anchor PMC text explicitly states sciatic nerve injection with AAV6 vectors carrying SwiChR-eYFP and analogous AAV6 constructs for hM4D(Gi), making AAV6 a key delivery component.

The Nature article explicitly states the ChRmine transgene was packaged in AAV9 for systemic cardiac delivery.

Subsequent evaluation of a codon-optimised microdystrophin transgene under the control of the optimal CAG promoter and capsid (AAV9K51Q) ... Based on the improved performance of AAV9K51Q vectors during intramuscular gene transfer, we performed a systemic administration of these vectors alone

AAV-based vectors-including AAV8 and synthetic AAVDJ-have demonstrated effective delivery of genes like Lhcgr into testicular interstitial tissues, restoring testosterone synthesis and fertility in mouse models.

Additionally, we describe the latest approaches for treatment, including AAV-mediated gene augmentation, genome editing, and late-stage therapies such as optogenetics, cell transplantation, and retinal prostheses.

Here, we show that a red-shifted channelrhodopsin (ReaChR), delivered by AAV injections in blind rd1 mice... We further show that postmortem macaque retinae infected with AAV-ReaChR can respond with spike trains to orange light at safe intensities.

Adeno-associated virus (AAV) vectors have become a cornerstone of in vivo gene delivery.

Seizure-like afterdischarges were successfully induced after the stimulation in both W-TChR2V4 transgenic (ChR2V-TG) rats and in wild type rats transfected with adeno-associated virus (AAV) vectors carrying ChR2.

MOR promoter (MORp) based constructs packaged into adeno-associated viral vectors

We injected adeno-associated virus vectors expressing the excitatory DREADD hM3Dq into the unilateral substantia nigra (SN) in four marmosets.

Adeno-associated virus vectors have emerged as the leading platform for inner ear gene transfer, owing to their safety and efficacy.

In this study, we implemented a fully non-invasive combined FUS-fUSi technique for performing optogenetics in mice.

Innovations in viral and non-viral delivery systems, such as dual AAV vectors, lipid nanoparticles, and novel biomaterials, have enhanced the efficiency and specificity of gene delivery to the retina.

Particular attention will be reserved to viral vectors derived from herpes simplex type 1, a potential tool for the delivery and expression of multiple transgene cassettes simultaneously.

The web research summary states that the anchor paper describes an adult human neocortical acute-and-cultured slice platform optimized for rapid molecular-genetic manipulation, emphasizing short-latency HSV-1 neuronal transduction, and lists HSV-1 amplicon vectors as the core genetic-delivery component.

lentiviral delivery of pegRNAs, ensuring robust, ubiquitous, and sustained expression of both prime editors and pegRNAs

Lentiviral vectors (LVVs) are used as a viral gene therapeutic and were derived from human immunodeficiency virus subtype 1 (HIV-1). LVVs are used to deliver and induce the stable expression of transgenes through genome integration.

Finally, to directly address the question of translatability to human subjects, we demonstrate for the first time, AAV- and lentivirus-mediated optogenetic spike responses in ganglion cells of the postmortem human retina.

Using a single-vector adeno-associated virus delivery system, we successfully induced REDMAPCre-mediated DNA recombination in mice.

increasing of expression of GluTs by viral gene transfer or positive pharmacological modulators can mitigate chronic pain

Expression of the chosen optogenetic tool in the cardiac cell of interest then requires, at the single-cell level, introduction of opsin-encoding genes by viral transduction.