| Rogers et al. |
Established a non-viral, antibody-based delivery method to transduce motor neurons in vivo after intraperitoneal injection. PEGylated polyethylenimine (PEI-PEG12) conjugated to a MRL2-antibody carrying DNA to the neurotrophin receptor p75 (p75NTR) targeted to motor neurons. 72 h after injection, ~25% of lumbar, ~18% or thoracic and 17% of cervical motor neurons were transduced. |
Wild-type mice |
| Smolny et al. |
Developed a non-viral, antibody-based delivery method for specific gene transfer in microglia in vitro and in vivo. OX42-immunoporter can bind plasmid DNA, and is trafficked to lysosomes in microglia via CD11b receptor-mediated internalization. OX42-immunogenes were specific to microglia and not astrocytes, but did not induce robust gene expression in vitro and in vivo. |
In vitro and Wild-type mice |
| Tanguy et al. |
Compared transduction efficiencies of scAAV9 and AAVrh10 in the brain, spinal, cord and peripheral nervous tissue after intravenous delivery in neonatal mice. AAVrh10 transduction was superior in the medulla, cerebellum, hippocampus, cortex, dorsal spinal cord, and spinal motor neurons. Dose-related transduction efficiency differences were observed in the sciatic nerve. |
Wild-type mice |
| Jackson et al. |
For the first time, AAV-PHP.B was demonstrated to transduce the rat CNS. After intravenous delivery in neonatal rats AAV-PHP.B was demonstrated to have a higher transduction efficiency than AAV9 when using the same CBA promoter. AAV-PHP.B with a synapsin promoter resulted in an enhanced transduction efficiency and neuronal specificity that induced TDP-43-like pathology and ALS-like phenotypes. |
Wild-type rats |
| von Jonquieres et al. |
Three MAG promoter sizes (0.3, 1.5, and 2.2 kb) were packaged into AAV-cy5 vector and were delivered into the striatum in wild-type neonates. All three promoter sizes exclusively transduced oligodendrocytes. Robust and oligodendrocyte-specific long-term GFP expression was reported at 8 months after neonatal delivery. |
In vitro and Wild-type mice |
|
Oliván et al. |
Application of a non-toxic, tetanus toxin fragment (TTC) to spinal cord organotypic cultures increased SMN levels. Intramuscular injections of TTC reduced mRNA of autophagy markers (Becn1, Atg5, LC3, and p62) and pro-apoptotic genes (Bax and Casp3) in the spinal cord and downregulated LC3 and Casp3 expression in skeletal muscle in SMA mice. Intramuscular TTC application is suggested to show a compensatory effect in the expression of certain genes involved in muscle damage response, oxidative stress and calcium homeostasis in SMA mice. |
Ex vivo and SMNΔ7 mice |
| Wu et al. |
Intraganglionic injections of AAV5-caRHEB into cervical DRGs transduced mainly large caliber DRG neurons. ChABC treatment increased the number of regenerating axons through the DREZ irrespective of DRG-transduction, which resulted in sensory behavioral “responses.” caRHEB expression in DRGs after dorsal root crush enhances synaptic formation and/or functional regeneration into the spinal gray matter. |
In vitro and Wild-type mice |
| Su et al. |
miR-30b agomir transfection down-regulated the voltage-gated sodium channel Nav1.3 mRNA that was stimulated with TNF-α in primary DRG neurons. miR-30b overexpression reduced neuropathic pain after spinal nerve ligation, with demonstrated reduction in Nav1.3 mRNA and protein expression in both DRG neurons and spinal cord. miR-30b antagomir activated the Nav1.3 voltage-gated sodium channel. |
In vitro and wild-type rats |
