Modeling Rett Syndrome with Stem Cells

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Modeling Rett Syndrome with Stem Cells

The discovery that somatic cells can be reprogrammed into induced pluripotent stem cells (iPSCs) raised the exciting possibility of modeling diseases with patient-specific cells. Marchetto et al. (2010) now use iPSC technology to generate, characterize, and treat an in vitro model for the autism spectrum disorder Rett syndrome.

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Neuronal maturation defect in induced pluripotent stem cells from patients with Rett syndrome.

Rett syndrome (RTT) is one of the most prevalent female neurodevelopmental disorders that cause severe mental retardation. Mutations in methyl CpG binding protein 2 (MeCP2) are mainly responsible for RTT. Patients with classical RTT exhibit normal development until age 6-18 mo, at which point they become symptomatic and display loss of language and motor skills, purposeful hand movements, and n...

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[Rett syndrome].

REFERENCES 1. Caldwell MB, Rogers MF. Epidemiology of pediatrics HIV infection. Pediatr Clin North Am 1991,38:1-16. 2. Centers for Disease Control. HIV/AIDS Surveillance Report. April, 1990. 3. Jovaisas E, Koch M, Schafer A, et al. LAV, HTLV III in 20 week fetus. Lancet 1985, 2:1129. 4. Zeigler JB, Cooper DA, Johnson RO, et al. Postnatal transmission of AIDSassociated retovirus from a mother to...

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Rett syndrome.

Rett syndrome is a progressive neurodegenerative disease of unknown etiology. Reported here are three children who presented with all clinical features of Rett syndrome. The aim of this presentation is to alert physicians to the existence of this progressive brain disease which only affects girls and so far no specific treatment has been suggested.

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Rett Syndrome

Rett syndrome is an X-linked neurological disorder that is a leading cause of mental retardation among females. The progression of the disease consists of normal neonatal development followed by a regression period during which neurodevelopmental signs such as mental retardation and decline of motor abilities start to appear. The developmental progress of Rett patients is within normal range fo...

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ژورنال

عنوان ژورنال: Cell

سال: 2010

ISSN: 0092-8674

DOI: 10.1016/j.cell.2010.10.037