@article{Prabhakar201918,
title = {Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1},
author = {S Prabhakar and P S Cheah and X Zhang and M Zinter and M Gianatasio and E Hudry and R T Bronson and D J Kwiatkowski and A Stemmer-Rachamimov and C A Maguire and M Sena-Esteves and B A Tannous and X O Breakefield},
url = {https://www.scopus.com/inward/record.uri?eid=2-s2.0-85070908794&doi=10.1016%2fj.omtm.2019.08.003&partnerID=40&md5=b169187dde0d3b05f8a9d5295a4ad8c4},
doi = {10.1016/j.omtm.2019.08.003},
issn = {23290501},
year = {2019},
date = {2019-01-01},
journal = {Molecular Therapy - Methods and Clinical Development},
volume = {15},
pages = {18-26},
publisher = {Cell Press},
abstract = {Tuberous sclerosis complex (TSC) is a tumor suppressor syndrome caused by mutations in TSC1 or TSC2, encoding hamartin and tuberin, respectively. These proteins act as a complex that inhibits mammalian target of rapamycin (mTOR)-mediated cell growth and proliferation. Loss of either protein leads to overgrowth in many organs, including subependymal nodules, subependymal giant cell astrocytomas, and cortical tubers in the human brain. Neurological manifestations in TSC include intellectual disability, autism, hydrocephalus, and epilepsy. In a stochastic mouse model of TSC1 brain lesions, complete loss of Tsc1 is achieved in homozygous Tsc1-floxed mice in a subpopulation of neural cells in the brain by intracerebroventricular (i.c.v.) injection at birth of an adeno-associated virus (AAV) vector encoding Cre recombinase. This results in median survival of 38 days and brain pathology, including subependymal lesions and enlargement of neuronal cells. Remarkably, when these mice were injected intravenously on day 21 with an AAV9 vector encoding hamartin, most survived at least up to 429 days in apparently healthy condition with marked reduction in brain pathology. Thus, a single intravenous administration of an AAV vector encoding hamartin restored protein function in enough cells in the brain to extend lifespan in this TSC1 mouse model. © 2019},
note = {cited By 2},
keywords = {Adeno Associated Virus, Adeno Associated Virus Vector, Animal Experiment, Animal Model, Article, Beta Actin, Blood Brain Barrier, Body Weight, Body Weight Gain, Brain Nerve Cell, Brain Ventricle, Cell Proliferation, Complementary DNA, Controlled Study, Cre Recombinase, Drug Efficacy, Female, Gene, Gene Replacement Therapy, Hamartin, HEK293 Cell Line, Hydrocephalus, Immunohistochemistry, Inverted Terminal Repeat, Long Term Care, Male, Motor Activity, Motor Performance, Mouse, Nonhuman, Priority Journal, Promoter Region, Protein Function, Protein Phosphorylation, Quantitative Analysis, Subventricular Zone, Survival Time, Tuberous Sclerosis, Tuberous Sclerosis Type 1, Vascularization, Viral Gene Delivery System},
pubstate = {published},
tppubtype = {article}
}

@article{Pandi-Perumal2007995,
title = {Role of the melatonin system in the control of sleep: Therapeutic implications},
author = {S R Pandi-Perumal and V Srinivasan and D W Spence and D P Cardinali},
url = {https://www.scopus.com/inward/record.uri?eid=2-s2.0-36248949004&doi=10.2165%2f00023210-200721120-00004&partnerID=40&md5=489ee976fa444beb95b26cdb77b722c2},
doi = {10.2165/00023210-200721120-00004},
issn = {11727047},
year = {2007},
date = {2007-01-01},
journal = {CNS Drugs},
volume = {21},
number = {12},
pages = {995-1018},
abstract = {The circadian rhythm of pineal melatonin secretion, which is controlled by the suprachiasmatic nucleus (SCN), is reflective of mechanisms that are involved in the control of the sleep/wake cycle. Melatonin can influence sleep-promoting and sleep/wake rhythm-regulating actions through the specific activation of MT1 (melatonin 1a) and MT2 (melatonin 1b) receptors, the two major melatonin receptor subtypes found in mammals. Both receptors are highly concentrated in the SCN. In diurnal animals, exogenous melatonin induces sleep over a wide range of doses. In healthy humans, melatonin also induces sleep, although its maximum hypnotic effectiveness, as shown by studies of the timing of dose administration, is influenced by the circadian phase. In both young and elderly individuals with primary insomnia, nocturnal plasma melatonin levels tend to be lower than those in healthy controls. There are data indicating that, in affected individuals, melatonin therapy may be beneficial for ameliorating insomnia symptoms. Melatonin has been successfully used to treat insomnia in children with attention-deficit hyperactivity disorder or autism, as well as in other neurodevelopmental disorders in which sleep disturbance is commonly reported. In circadian rhythm sleep disorders, such as delayed sleep-phase syndrome, melatonin can significantly advance the phase of the sleep/wake rhythm. Similarly, among shift workers or individuals experiencing jet lag, melatonin is beneficial for promoting adjustment to work schedules and improving sleep quality. The hypnotic and rhythm-regulating properties of melatonin and its agonists (ramelteon, agomelatine) make them an important addition to the armamentarium of drugs for treating primary and secondary insomnia and circadian rhythm sleep disorders. © 2007 Adis Data Information BV. All rights reserved.},
note = {cited By 90},
keywords = {Absence of Side Effects, Acetylserotonin Methyltransferase, Advanced Sleep Phase Syndrome, Agomelatine, Alpha Tocopherol, Alzheimer Disease, Animals, Ascorbic Acid, Beta Adrenergic Receptor Blocking Agent, Biosynthesis, Circadian Rhythm, Circadian Rhythm Sleep Disorder, Clinical Trial, Confusion, Delayed Sleep Phase Syndrome, Drowsiness, Drug Dose Comparison, Drug Efficacy, Drug Half Life, Drug Mechanism, Fatigue, Fluvoxamine, Headache, Hormone Metabolism, Human, Hypnosis, Hypothalamus, Insomnia, Jet Lag, Macaca, Melatonin, Melatonin Receptor, Muscle Cramp, Nausea, Non-24-Hour Sleep-Wake Syndrome, Nonhuman, Noradrenalin, Pineal Body, Priority Journal, Protein Expression, Ramelteon, Rat Strain, Receptor Density, Receptors, REM Sleep, Retina Ganglion Cell, Review, Serotonin, Shift Worker, Sleep, Sleep Disorder, Sleep Waking Cycle, Smith Magenis Syndrome, Suprachiasmatic Nucleus, Sustained Drug Release, Vomiting},
pubstate = {published},
tppubtype = {article}
}

@article{Jayachandra200599,
title = {Is secretin effective in treatment for Autism Spectrum Disorders (ASD)?},
author = {S Jayachandra},
url = {https://www.scopus.com/inward/record.uri?eid=2-s2.0-23044481281&doi=10.2190%2fQ1D2-5DNB-V4FJ-J9M5&partnerID=40&md5=791bd90c9cdaa7b82bc5d7e8b8c5a7de},
doi = {10.2190/Q1D2-5DNB-V4FJ-J9M5},
issn = {00912174},
year = {2005},
date = {2005-01-01},
journal = {International Journal of Psychiatry in Medicine},
volume = {35},
number = {1},
pages = {99-101},
note = {cited By 2},
keywords = {Autism, Autism Spectrum Disorders, Children, Clinical Trial, Drug Effect, Drug Efficacy, Drug Mechanism, Gastrointestinal Symptom, Human, Hypersensitivity, Injections, Intravenous, Letter, Secretin, Treatment Outcome},
pubstate = {published},
tppubtype = {article}
}