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. Author manuscript; available in PMC: 2015 Nov 1.

Published in final edited form as: Pediatr Neurol. 2014 Aug 8;51(5):600–606. doi: 10.1016/j.pediatrneurol.2014.08.003

Table 1.

Single Therapies for Murine GLD

Therapy	Putative Mechanism	Lifespan of Treated GALC−/− Mutants	References
Bone Marrow Transplant	Cross-Correction Immunomodulation	GALC+/+ Donor: 80 days^*	8, 9, 12, 13
Bone Marrow Transplant	Cross-Correction Immunomodulation	GALC-Overexpressing GALC−/− Donor: 88 days^*	14
Mesenchymal Stem Cells	Immunomodulation Replacement of damaged neuronal cells	40 days	23–27
Enzyme Replacement Therapy	Short-term replacement of deficient GALC activity	Single-dose ICV administration: 50 days^*	31
Enzyme Replacement Therapy	Short-term replacement of deficient GALC activity	Weekly IP administration: 47 days^*	35
Viral-Mediated Gene Therapy	Long-term replacement of deficient GALC activity	AAV2/5-GALC IC administration: 63 days	40
		AAV2/5-GALC IC+IT administration: 71 days	10
		AAVrh10-GALC adminsitration: 120 days^*	42
NSC-Directed Gene Therapy	Cross-Correction Replacement of damaged neuronal cells	Recombinant retroviral-GALC transduced NSCs: 45 days	29
NSC-Directed Gene Therapy	Cross-Correction Replacement of damaged neuronal cells	Recombinant lentiviral-GALC transduced NSCs: 53 days^*	30
L-Cycloserine	Substrate Reduction Therapy	57 days^*	45
Anti-Inflammatory Therapies	Immunomodulation	Ibudilast: Not reported^**	52
Anti-Inflammatory Therapies	Immunomodulation	Indomethacin: 65 days	53

^*

Mean lifespans. All other life spans are represented by the median.

^**

Exact lifespan of treated Twi mice not reported, but was not significantly different compared to untreated mutants