Experimental neurotransplantation treatment for hereditary cerebellar ataxias

Cendelin, Jan

doi:10.1186/s40673-016-0045-3

Cerebellum & Ataxias

Table 1 Experimental therapeutic approaches for cerebellar degenerations

From: Experimental neurotransplantation treatment for hereditary cerebellar ataxias

Therapy	Subject	Effect	References
Inhibition of mutated gene expression	SCA3 mice	-clearance of nuclear accumulation of ataxin-3 -improved motor performance -alleviation of neuropathological changes	[14]
	SCA3 mice	-suppression of already manifested symptoms	[11]
	SCA7 mice	-reduction of ataxin-7 aggregation -improved motor performance -prevention of synaptic loss between climbing fibers and Purkinje cells	[13]
Enhancement of pathological protein elimination	SCA3 mice	-decrease of ataxin-3 accumulation -suppression of pontine neuronal death -improved motor performance	[15]
Enhancement of pathological protein elimination	SCA7 mice	-reduction of ataxin-7 intranuclear inclusions -improved motor performance	[12]
Stabilization of calcium signaling	SCA2, SCA3 mice	-alleviation of neuronal cell loss -improved motor performance	[17, 18]
Transcranial direct current stimulation	Human patients	-neuromodulation of the cerebellum	[19]
Embryonic (fetal) neural tissue transplantation	pcd mice	-colonization of the host molecular layer	[31–33, 35, 38]
	pcd mice	-improved motor performance	[40, 41]
	Lurcher mice	-organotypic organization of the graft, colonization of the host molecular layer	[54, 58, 59]
	Lurcher mice	-mild improvement of gait	[57]
	Weaver mice	-organotypic organization of the graft -colonization of the host cerebellum with granule-like cells	[64, 65]
	SCA1 mice	-improved motor performance	[80]
Neural precursor transplantation	Nervous mice	-support and rescue of host’s Purkinje cells -reduction of the excessive tissue plasminogen activator level -improved motor performance	[71, 72]
	Meander tail mice	-replenishment of deficient granule cell population	[76]
	SCA1 mice	-increased survival of Purkinje cells -improved motor performance	[81]
	SCA3 mice	-increased Purkinje cell survival -reduced granular layer atrophy -improved motor performance	[85]
	Niemann-Pick disease type C mice	-lengthened their life span without influencing the decline of motor performance	[92]
	Niemann-Pick disease type C mice	-increased Purkinje cell survival -alleviation of neuropathological changes	[93]
	Niemann-Pick disease type A mice	-alleviation of neuropathological changes without influencing the decline of motor performance	[86]
Embryonic stem cell, embryonic stem cell-derived neural precursor transplantation	Weaver mice	-no adoption of region-specific cell identities	[67]
	Harlequin mice	-induction of endogenous neuronal precursor proliferation -delayed onset of the ataxia	[79]
Mesenchymal stem cell transplantation	Lurcher mice	-increased Purkinje cell survival -improved motor performance	[60]
	SCA1 mice	-mitigated cerebellar disorganization -improved motor performance	[82]
	SCA2 mice	-increased Purkinje cell survival -delayed disease onset -improved motor performance	[83]
	Niemann-Pick disease type C mice	-reduced astrocytic and microglial activation -increased Purkinje cell survival -improved motor performance	[87–90]

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ISSN: 2053-8871

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