Gangliosides as Therapeutic Targets for Neurodegenerative Diseases

Table 2

In vivo ganglioside-based treatment of neurodegenerative disorders which affect the neuropathology of these disorders.

Disorder	Species	Ganglioside	Route	Clinical signs	References
AD	Human	GM1 ➔ 24 weeks	Intramuscular	No overall symptomatic benefit	[28]
AD	Human	GM1 ➔ 6 weeks	Intramuscular	No improvement in cognitive function.	[29]
AD	Human	GM1 ➔ 12 months	Intrathecal	Disease pathology is halted by continuous treatment.	[30]
AD	Human	GM1	Intracerebroventricular	Improvement of physical activities	[31, 32]
AD	Mouse (APP/PS1)	GM1	Intraperitoneal	Reduction of Aβ-40 and Aβ-42.	[33]
AD	Mouse (Tg2576)	4396C	Intraperitoneal	Reduction of Aβ-40 and Aβ-42 deposits.	[34]
AD	Mouse (APP/PS1)	GM1	Intraperitoneal	Exacerbating cognitive dysfunction	[35, 36]
AD	Mouse (APP/PS1)	AAV-NEU3 (overexpression)	Intracerebroventricular	Accumulation of the Aβ deposits.	[35]
AD	Mouse (APP/PS1)	D-PDMP	Intraperitoneal	Reduction of Aβ deposits and rescue memory.	[35]
AD	Mouse (Tg2576)	GCSi (GENZ 667161)	Diet	Mitigation of the soluble Aβ-42 and amyloid plaque burden.	[37]
AD	Mouse (3xTg-AD)	GQ1b	Intrahippocampal	Reduction of Aβ plaque deposition and tau phosphorylation.	[38]
PD	Mouse (B4galnt1+/-)	LIGA-20	Intraperitoneal	Reduced TH^+ neuron cell death and αSyn accumulation	[11]
PD	Mouse (B4galnt1-/-)	LIGA-20	Intraperitoneal	Reduced αSyn accumulation	[21]
PD	Rat (overexpressing human mutant A53T αSyn)	GM1	Intraperitoneal	Protection against striatal dopamine depletion and dopaminergic neuron death	[39]
PD	Mouse (B4galnt1+/-)	OligoGM1 (GM1-OS)	Intraperitoneal	Reduced αSyn accumulation in SN, increased tyrosine hydroxylase	[40]
PD	Human	GM1 (combined with pramipexole)	Intravenous	Decreased inflammatory CRP and TNF-α levels, elevated motor functions	[41]
PD	Mouse (overexpressing human mutant A53T αSyn)	GM1 and GD3	Intranasal	Induced neurogenesis and promoted self-renewal ability of neural stem cells	[42]
HD	Mouse (R6/2, Q140, YAC128)	GM1	Intraventricular	Reduced motor defects, neurodegeneration, and huntingtin levels	[43] [44]
ALS	Mouse SOD1^G86R and SOD1^G93A	IgM (rHIgM12) targeting GD1a and GT1b	Intraperitoneal	Prolonged survival and slowed neurological impairments	[45]