Tuberous sclerosis complex

EP Henske, S Jóźwiak, JC Kingswood… - Nature reviews Disease …, 2016 - nature.com
Tuberous sclerosis complex (TSC) is an autosomal dominant disorder that affects multiple
organ systems and is caused by loss-of-function mutations in one of two genes: TSC1 or …

[HTML][HTML] Molecular neurobiology of mTOR

K Switon, K Kotulska, A Janusz-Kaminska… - Neuroscience, 2017 - Elsevier
Mammalian/mechanistic target of rapamycin (mTOR) is a serine-threonine kinase that
controls several important aspects of mammalian cell function. mTOR activity is modulated …

[HTML][HTML] Updated international tuberous sclerosis complex diagnostic criteria and surveillance and management recommendations

H Northrup, ME Aronow, EM Bebin, J Bissler… - Pediatric Neurology, 2021 - Elsevier
Background Tuberous sclerosis complex (TSC) is an autosomal dominant genetic disease
affecting multiple body systems with wide variability in presentation. In 2013, Pediatric …

Somatic mutations activating the mTOR pathway in dorsal telencephalic progenitors cause a continuum of cortical dysplasias

AM D'Gama, MB Woodworth, AA Hossain, S Bizzotto… - Cell reports, 2017 - cell.com
Focal cortical dysplasia (FCD) and hemimegalencephaly (HME) are epileptogenic
neurodevelopmental malformations caused by mutations in mTOR pathway genes. Deep …

Epilepsy in the mTORopathies: opportunities for precision medicine

PB Moloney, GL Cavalleri, N Delanty - Brain communications, 2021 - academic.oup.com
The mechanistic target of rapamycin signalling pathway serves as a ubiquitous regulator of
cell metabolism, growth, proliferation and survival. The main cellular activity of the …

Somatic overgrowth disorders of the PI3K/AKT/mTOR pathway & therapeutic strategies

KM Keppler‐Noreuil, VER Parker… - American Journal of …, 2016 - Wiley Online Library
The phosphatidylinositol‐3‐kinase (PI3K)/AKT/mTOR signaling pathway plays an essential
role in regulation of normal cell growth, metabolism, and survival. Somatic activating …

Genetically engineered human cortical spheroid models of tuberous sclerosis

JD Blair, D Hockemeyer, HS Bateup - Nature medicine, 2018 - nature.com
Tuberous sclerosis complex (TSC) is a multisystem developmental disorder caused by
mutations in the TSC1 or TSC2 genes, whose protein products are negative regulators of …

Genetic etiologies, diagnosis, and treatment of tuberous sclerosis complex

CL Salussolia, K Klonowska… - Annual Review of …, 2019 - annualreviews.org
Tuberous sclerosis complex (TSC) is an autosomal dominant disorder that affects multiple
organ systems due to an inactivating variant in either TSC1 or TSC2, resulting in the …

TuberOus SClerosis registry to increase disease Awareness (TOSCA)–baseline data on 2093 patients

JC Kingswood, GB d'Augères, E Belousova… - Orphanet journal of rare …, 2017 - Springer
Background Tuberous sclerosis complex (TSC) is a rare autosomal dominant genetic
disorder. Many gaps remain in the understanding of TSC because of the complexity in …

The genomic landscape of tuberous sclerosis complex

KR Martin, W Zhou, MJ Bowman, J Shih, KS Au… - Nature …, 2017 - nature.com
Tuberous sclerosis complex (TSC) is a rare genetic disease causing multisystem growth of
benign tumours and other hamartomatous lesions, which leads to diverse and debilitating …