Table 3.

Lentiviral vectors for brain-targeted gene delivery

Gene	Disease/Mechanism	Transduced Cell Types	In Vitro Cell Source	In Vivo	Result	Reference
CNTF	Excitotoxicity	Neurons	SD rats	Quinolinic acid (QA)-induced Lewis rats	Hypertrophied astrocytes with increased glutamate aspartate transporter (GLAST) and glutamate transporter-1 (GLT-1) expression, improved glutamate handling	(Escartin et al., 2006)
GDNF	NDDs	Neurons, Astrocytes	Mouse N2a neural cells, ventral mesencep halon cells		Gene expression for 3+ weeks, neuroprotection from 6-OHDA insult mediated by GDNF and glutathione together	(Sandhu et al., 2009)
GDNF	PD	Astrocytes		6-OHDA treated SD rats	Efficient, sustained expression, improved DA neuronal survival, improved behavior	(Lu-Nguyen et al., 2014)
GFP	PD	Neurons, Astrocytes		Lewis rats	Astrocyte tropism by lymphocytic choriomeningitis virus (LCMV)- and Moloney murine leukemia virus (muLV)-enveloped	(Cannon et al., 2011)
GFP	Neural scar formation	Neurons, Astrocytes, Oligodendrocytes precursors, Microglia	Wistar rats	Wistar rats	In vitro astrocyte and meningeal cells transduction, lack of transduction at injury/injection site in vivo with peripheral transduction of several cell types except meningeal cells	(Hendriks et al., 2007)
GFP		Neurons, Astrocytes		B6 mice	Astrocytes transduced with neuron promoter	(Lai and Brady, 2002)
GFP		Neurons, Astrocytes		SD rats	Vesicular stomatitis virus glycoprotein (VSV-G)-pseudotyped vector transduces glia and neurons, promoter-dependent selectivity	(Jakobsson et al., 2003)
GFP		Neurons, Astrocytes		SD rats, MF1 mice	Astrocyte tropism by gp64-enveloped	(Rahim et al., 2009)
GFP		Neurons, Astrocytes		Cynomolgus monkeys	Higher astrocyte transduction without activation over AAV2	(An et al., 2016)
LacZ	Demyelination	Astrocytes, Oligodendrocytes, Oligodendrocyte progenitors		SD rats	Expressed in glial cells of spinal cord white matter, microglia and macrophages at injection site	(Zhao et al., 2003)
LacZ		Neurons, Astrocytes, Microglia	SD rats	C3H mice	Different brain regions transduced by muLV, LCMV, Mokola virus, VSV-G enveloped, lack of transduction with Ebola virus enveloped	(Watson et al., 2002)
LacZ		Neurons		Fisher rats	Neurons ~90% of transduced cells at the injection site	(Blömer et al., 1997)
miR124, shRNA GLAST, dsRednuc, mCherry		Astrocytes		BAC-GLT1-eGFP, BAC-GLAST-DsRed, B6 mice	Neuron-specific silencing, used other miRNAs; astrocyte-specific promoters and tetracycline-inducible systems for astrocyte-targeting and regulation	(Merienne et al., 2015)
miR124T, GLAST, LacZ		Neurons, Astrocytes	SD rats	B6 mice	miR124T blocked neuronal expression and reporter expressed in astrocytes	(Colin et al., 2009)
miRNA chondroitin polymerizing factor or chondroitin synthase-1		Astrocytes	Neu7 rats, chicken embryo dorsal root ganglia		Reduced chondroitin sulphate proteoglycans and neurite outgrowth inhibition	(Tuinstra et al., 2013)
Neurotro phin-3, BDNF	SCI	Astrocytes, Schwann cells		Long-Evans rats	Transduced multiple cell types, increased axonal growth and myelination	(Tuinstra et al., 2012)
Small hairpin RNA (shRNA) GFAP, vimentin	SCI	Astrocytes	B6 mice neuroglia		Reduced glial scarring, improved neuronal survival; axonal growth increased or not affected by shGFAP	(Desclaux et al., 2009)