. Author manuscript; available in PMC: 2011 May 4.

Published in final edited form as: Nat Rev Neurosci. 2010 Jul;11(7):490–502. doi: 10.1038/nrn2851

Table 2.

Examples of treatments that prevented or reversed phenotypes in mouse models of neurodevelopmental disorders

Treatment	Mouse model	Phenotypic improvement
mGluR antagonists, MPEP^88,161,162, fenobam¹⁶²	Fmr1 ^−/−	Susceptibility to audiogenic seizures is prevented¹⁶¹ Decreased open field hyperactivity¹⁶¹ Rescued prepulse inhibition of startle deficit¹⁶² Rescued abnormal spine morphology¹⁶²
mGluR antagonists, MPEP^88,161,162, fenobam¹⁶²	BTBR	Reduced repetitive behaviour⁸⁸
mTOR inhibitors, rapamycin^{63,77,177,178}, RAD001 (Ref. 177)	Pten	Prevented and reversed macrocephaly, dendritic and axonal hypertrophy⁷⁷ Improved social interaction time⁷⁷ Increased open field centre time⁷⁷ Reduced duration and frequency of seizures⁷⁷
	Tsc1 null-neuron inactivated in neurons^63,177	Improved survival rates^63,177 Improved neuronal morphology, reduced enlarged neurons and restored myelination¹⁷⁷
	Tsc1^GFAP inactivated in glia¹⁷⁸	Improved survival rates and weight gain¹⁷⁸ Prevented seizures and electroencephalography(EEG) abnormalities¹⁷⁸
	Tsc2^+/− (Ref. 63)	Improved learning and memory on Morris water maze and fear conditioning⁶³
Oxytocin¹¹⁴	OXT ^−/−	Rescued deficits in social recognition¹¹⁴
BDNF⁷⁵	Fmr1 ^−/−	Rescued long-term potentiation abnormality⁷⁵
Ampakines, CX546 (Ref. 73)	Mecp2 ^−/−	Reversed respiratory deficits⁷³
mGluR genetic reduction⁷⁴	Fmr1 ^−/−	Prevented susceptibility to audiogenic seizures⁷⁴ Rescued abnormal spine morphology⁷⁴ Rescue of exaggerated inhibitory avoidance learning⁷⁴
FMR1 gene replacement^60,61,76	Fmr1 ^−/−	Normalized open field activity⁶⁰ Normalized light–dark anxiety-like behaviour⁶⁰ Rescued abnormal social responses⁶¹ Rescued increased prepulse inhibition⁷⁶
PAK genetic reduction⁹²	Fmr1 ^−/−	Normalized open field centre time⁹² Rescued fear-conditioning deficit⁹² Rescued long-term potentiation deficit⁹²
MECP2 gene replacement^174,176	Mecp2^−/+ is an inducible heterozygous transgenic¹⁷⁶	Rescued open field deficits¹⁷⁶ Increased survival and lifespan¹⁷⁴
MECP2 gene replacement^174,176	Mecp2/Stop is an Mecp2 mutant with Mecp2 conditional activation¹⁷⁴	Normalized weights, breathing, gait and activity¹⁷⁴

See TABLE 1 and main text for further details on mouse models. AMPA,α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid; BDNF, brain-derived neurotrophic factor; Fmr1, fragile × mental retardation syndrome 1; Mecp2, methyl-CpG-binding protein 2; mGluR, metabotropic glutamate receptor; MPEP, 2-methyl-6-phenylethynyl-pyridine hydrochloride; mTOR, mammalian target of rapamycin; PAK, p21-activated kinase; Pten, phosphatase and tensin homologue; Tsc, tuberous sclerosis.