Drug discovery for autism spectrum disorder: challenges and opportunities
Abstract
The rising rates of autism spectrum disorder (ASD) and the lack of effective medications to treat its core symptoms have led to an increased sense of urgency to identify therapies for this group of neurodevelopmental conditions. Developing drugs for ASD, however, has been challenging because of a limited understanding of its pathophysiology, difficulties in modelling the disease in vitro and in vivo, the heterogeneity of symptoms, and the dearth of prior experience in clinical development. In the past few years these challenges have been mitigated by considerable advances in our understanding of forms of ASD caused by single-gene alterations, such as fragile X syndrome and tuberous sclerosis. In these cases we have gained insights into the pathophysiological mechanisms underlying these conditions. In addition, they have aided in the development of animal models and compounds with the potential for disease modification in clinical development. Moreover, genetic studies are illuminating the molecular pathophysiology of ASD, and new tools such as induced pluripotent stem cells offer novel possibilities for drug screening and disease diagnostics. Finally, large-scale collaborations between academia and industry are starting to address some of the key barriers to developing drugs for ASD. Here, we propose a conceptual framework for drug discovery in ASD encompassing target identification, drug profiling and considerations for clinical trials in this novel area.
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Developmental anomalies and behavioral deficits observed during childhood may be, at least partially, modifiable.
“promising findings suggest that developmental anomalies and behavioral deficits observed during childhood may be, at least partially, modifiable [53]”
From Prevention and early intervention in youth mental health: is it time for a multidisciplinary and trans-diagnostic model for care? (2020) · cites this paper as [53]
Cites
(1943) · The Nervous child · Cited by 8,870 · see citing works
Finding the missing heritability of complex diseases
Manolio, T. A., Collins, F. S., Cox, N. J., Goldstein, D. B., Hindorff, L. A., Hunter, D. J., McCarthy, M. I., Ramos, E. M., Cardon, L. R., Chakravarti, A., et al. (2009) · Nature · doi:10.1038/nature08494 · Cited by 8,336 · see citing works
Network medicine: a network-based approach to human disease
Barabási, A.-L., Gulbahce, N., & Loscalzo, J. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2918 · Cited by 5,034 · see citing works
STRING v9.1: protein-protein interaction networks, with increased coverage and integration
Franceschini, A., Szklarczyk, D., Frankild, S., Kuhn, M., Simonovic, M., Roth, A., Lin, J., Minguez, P., Bork, P., von Mering, C., et al. (2012) · Nucleic Acids Research · doi:10.1093/nar/gks1094 · Cited by 4,398 · see citing works
Simonoff, E., Pickles, A., Charman, T., Chandler, S., Loucas, T., & Baird, G. (2008) · Journal of the American Academy of Child & Adolescent Psychiatry · doi:10.1097/chi.0b013e318179964f · Cited by 3,399 · see citing works
Genome-wide association studies for complex traits: consensus, uncertainty and challenges
McCarthy, M. I., Abecasis, G. R., Cardon, L. R., Goldstein, D. B., Little, J., Ioannidis, J. P. A., & Hirschhorn, J. N. (2008) · Nature Reviews Genetics · doi:10.1038/nrg2344 · Cited by 2,891 · see citing works
Rate of de novo mutations and the importance of father’s age to disease risk
Kong, A., Frigge, M. L., Masson, G., Besenbacher, S., Sulem, P., Magnusson, G., Gudjonsson, S. A., Sigurdsson, A., Jonasdottir, A., Jonasdottir, A., et al. (2012) · Nature · doi:10.1038/nature11396 · Cited by 2,134 · see citing works
FMRP Stalls Ribosomal Translocation on mRNAs Linked to Synaptic Function and Autism
Darnell, J. C., Van Driesche, S. J., Zhang, C., Hung, K. Y. S., Mele, A., Fraser, C. E., Stone, E. F., Chen, C., Fak, J. J., Chi, S. W., et al. (2011) · Cell · doi:10.1016/j.cell.2011.06.013 · Cited by 2,072 · see citing works
Epidemiology of Pervasive Developmental Disorders
Fombonne, E. (2009) · Pediatric Research · doi:10.1203/pdr.0b013e31819e7203 · Cited by 1,971 · see citing works
Dimos, J. T., Rodolfa, K. T., Niakan, K. K., Weisenthal, L. M., Mitsumoto, H., Chung, W. K., Croft, G. F., Saphier, G., Leibel, R. L., Goland, R., et al. (2008) · Science · doi:10.1126/science.1158799 · Cited by 1,959 · see citing works
Patterns and rates of exonic de novo mutations in autism spectrum disorders
Neale, B. M., Kou, Y., Liu, L., Ma’ayan, A., Samocha, K. E., Sabo, A., Lin, C.-F., Stevens, C., Wang, L.-S., Makarov, V., et al. (2012) · Nature · doi:10.1038/nature11011 · Cited by 1,734 · see citing works
A travel guide to Cytoscape plugins
Saito, R., Smoot, M. E., Ono, K., Ruscheinski, J., Wang, P.-L., Lotia, S., Pico, A., Bader, G. D., & Ideker, T. (2012) · Nature Methods · doi:10.1038/nmeth.2212 · Cited by 1,511 · see citing works
Behavioural phenotyping assays for mouse models of autism
Silverman, J. L., Yang, M., Lord, C., & Crawley, J. N. (2010) · Nature Reviews Neuroscience · doi:10.1038/nrn2851 · Cited by 1,462 · see citing works
Marchetto, M. C. N., Carromeu, C., Acab, A., Yu, D., Yeo, G. W., Mu, Y., Chen, G., Gage, F. H., & Muotri, A. R. (2010) · Cell · doi:10.1016/j.cell.2010.10.016 · Cited by 1,329 · see citing works
Rare and common variants: twenty arguments
Gibson, G. (2012) · Nature Reviews Genetics · doi:10.1038/nrg3118 · Cited by 1,278 · see citing works
Sirolimus for Angiomyolipoma in Tuberous Sclerosis Complex or Lymphangioleiomyomatosis
Bissler, J. J., McCormack, F. X., Young, L. R., Elwing, J., Chuck, G., Leonard, J. M., Schmithorst, V. J., Laor, T., Brody, A. S., Bean, J. A., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa063564 · Cited by 1,250 · see citing works
Curatolo, P., Bombardieri, R., & Jóźwiak, S. (2008) · The Lancet · doi:10.1016/s0140-6736(08)61279-9 · Cited by 1,106 · see citing works
Correction of Fragile X Syndrome in Mice
Dölen, G., Osterweil, E., Rao, B. S. S., Smith, G. B., Auerbach, B. D., Chattarji, S., & Bear, M. F. (2007) · Neuron · doi:10.1016/j.neuron.2007.12.001 · Cited by 979 · see citing works
Betancur, C. (2011) · Brain Research · doi:10.1016/j.brainres.2010.11.078 · Cited by 954 · see citing works
Promoting social behavior with oxytocin in high-functioning autism spectrum disorders.
Andari, E., Duhamel, J.-R., Zalla, T., Herbrecht, E., Leboyer, M., & Sirigu, A. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0910249107 · Cited by 941 · see citing works
Reversal of learning deficits in a Tsc2+/− mouse model of tuberous sclerosis
Ehninger, D., Han, S., Shilyansky, C., Zhou, Y., Li, W., Kwiatkowski, D. J., Ramesh, V., & Silva, A. J. (2008) · Nature Medicine · doi:10.1038/nm1788 · Cited by 879 · see citing works
Buie, T., Campbell, D. B., Fuchs, G. J., Furuta, G. T., Levy, J., VandeWater, J., Whitaker, A. H., Atkins, D., Bauman, M. L., Beaudet, A. L., et al. (2010) · Pediatrics · doi:10.1542/peds.2009-1878c · Cited by 872 · see citing works
GSK3 signalling in neural development
Hur, E., & Zhou, F. (2010) · Nature reviews. Neuroscience · doi:10.1038/nrn2870 · Cited by 828 · see citing works
Autism‐like behavioral phenotypes in BTBR T+tf/J mice
McFarlane, H. G., Kusek, G. K., Yang, M., Phoenix, J. L., Bolivar, V. J., & Crawley, J. N. (2008) · Genes, Brain and Behavior · doi:10.1111/j.1601-183x.2007.00330.x · Cited by 812 · see citing works
Pervasive Developmental Disorders in Preschool Children: Confirmation of High Prevalence
Chakrabarti, S., & Fombonne, E. (2005) · American Journal of Psychiatry · doi:10.1176/appi.ajp.162.6.1133 · Cited by 791 · see citing works
Phenotypic Heterogeneity of Genomic Disorders and Rare Copy-Number Variants
Girirajan, S., Rosenfeld, J. A., Coe, B. P., Parikh, S., Friedman, N., Goldstein, A., Filipink, R. A., McConnell, J. S., Angle, B., Meschino, W. S., et al. (2012) · New England Journal of Medicine · doi:10.1056/nejmoa1200395 · Cited by 621 · see citing works
Autism spectrum disorders and autistic traits: A decade of new twin studies
Ronald, A., & Hoekstra, R. A. (2011) · American Journal of Medical Genetics Part B: Neuropsychiatric Genetics · doi:10.1002/ajmg.b.31159 · Cited by 616 · see citing works
Jacquemont, S., Curie, A., des Portes, V., Torrioli, M. G., Berry-Kravis, E., Hagerman, R. J., Ramos, F. J., Cornish, K., He, Y., Paulding, C., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3001708 · Cited by 609 · see citing works
The Autistic Neuron: Troubled Translation?
Kelleher, R. J., & Bear, M. F. (2008) · Cell · doi:10.1016/j.cell.2008.10.017 · Cited by 598 · see citing works
Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice
Tropea, D., Giacometti, E., Wilson, N. R., Beard, C., McCurry, C., Fu, D. D., Flannery, R., Jaenisch, R., & Sur, M. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0812394106 · Cited by 593 · see citing works
Mutations causing syndromic autism define an axis of synaptic pathophysiology
Auerbach, B. D., Osterweil, E. K., & Bear, M. F. (2011) · Nature · doi:10.1038/nature10658 · Cited by 592 · see citing works
Mechanism for the learning deficits in a mouse model of neurofibromatosis type 1
Costa, R. M., Federov, N. B., Kogan, J. H., Murphy, G. G., Stern, J. N. H., Ohno, M., Kucherlapati, R., Jacks, T., & Silva, A. J. (2002) · Nature · doi:10.1038/nature711 · Cited by 586 · see citing works
Using iPSC-derived neurons to uncover cellular phenotypes associated with Timothy syndrome
Paşca, S. P., Portmann, T., Voineagu, I., Yazawa, M., Shcheglovitov, A., Paşca, A. M., Cord, B., Palmer, T. D., Chikahisa, S., Nishino, S., et al. (2011) · Nature Medicine · doi:10.1038/nm.2576 · Cited by 585 · see citing works
Whole-Genome Sequencing in Autism Identifies Hot Spots for De Novo Germline Mutation
Michaelson, J. J., Shi, Y., Gujral, M., Zheng, H., Malhotra, D., Jin, X., Jian, M., Liu, G., Greer, D., Bhandari, A., et al. (2012) · Cell · doi:10.1016/j.cell.2012.11.019 · Cited by 544 · see citing works
Meikle, L. M., Pollizzi, K. N., Egnor, A., Kramvis, I., Lane, H. A., Şahin, M., & Kwiatkowski, D. J. (2008) · Journal of Neuroscience · doi:10.1523/jneurosci.0955-08.2008 · Cited by 500 · see citing works
Regulation of neuronal morphology and function by the tumor suppressors Tsc1 and Tsc2
Tavazoie, S. F., Alvarez, V. A., Ridenour, D. A., Kwiatkowski, D. J., & Sabatini, B. L. (2005) · Nature Neuroscience · doi:10.1038/nn1566 · Cited by 497 · see citing works
Chronic Pharmacological mGlu5 Inhibition Corrects Fragile X in Adult Mice
Michalon, A., Sidorov, M., Ballard, T. M., Ozmen, L., Spooren, W., Wettstein, J. G., Jaeschke, G., Bear, M. F., & Lindemann, L. (2012) · Neuron · doi:10.1016/j.neuron.2012.03.009 · Cited by 489 · see citing works
Self-citationBilousova, T. V., Dansie, L., Ngo, M., Aye, J., Charles, J. R., Ethell, D. W., & Ethell, I. M. (2009) · Journal of Medical Genetics · doi:10.1136/jmg.2008.061796 · Cited by 465 · see citing works
Meikle, L., Talos, D. M., Onda, H., Pollizzi, K., Rotenberg, A., Sahin, M., Jensen, F. E., & Kwiatkowski, D. J. (2007) · The Journal of Neuroscience · doi:10.1523/jneurosci.5540-06.2007 · Cited by 462 · see citing works
In utero exposure to valproic acid and autism — A current review of clinical and animal studies
Roullet, F., Lai, J., & Foster, J. A. (2013) · Neurotoxicology and Teratology · doi:10.1016/j.ntt.2013.01.004 · Cited by 429 · see citing works
Common genetic variants, acting additively, are a major source of risk for autism
Klei, L., Sanders, S. J., Murtha, M. T., Hus, V., Lowe, J. K., Willsey, A. J., Moreno-De-Luca, D., Yu, T. W., Fombonne, E., Geschwind, D., et al. (2012) · Molecular Autism · doi:10.1186/2040-2392-3-9 · Cited by 418 · see citing works
Mattila, M., Hurtig, T. M., Haapsamo, H., Jussila, K. K., Kuusikko‐Gauffin, S., Kielinen, M., Linna, S., Ebeling, H. E., Bloigu, R., Joskitt, L., et al. (2010) · Journal of Autism and Developmental Disorders · doi:10.1007/s10803-010-0958-2 · Cited by 392 · see citing works
Genetic and Epigenetic Networks in Intellectual Disabilities
van Bokhoven, H. (2011) · Annual Review of Genetics · doi:10.1146/annurev-genet-110410-132512 · Cited by 383 · see citing works
The factor structure of autistic traits
Constantino, J. N., Gruber, C. P., Davis, S., Hayes, S., Passanante, N., & Przybeck, T. (2004) · Journal of Child Psychology and Psychiatry · doi:10.1111/j.1469-7610.2004.00266.x · Cited by 370 · see citing works
Sensory features and repetitive behaviors in children with autism and developmental delays
Boyd, B. A., Baranek, G. T., Sideris, J. H., Poe, M. D., Watson, L. R., Patten, E., & Miller, H. (2010) · Autism Research · doi:10.1002/aur.124 · Cited by 358 · see citing works
Etherton, M. R., Földy, C., Sharma, M., Tabuchi, K., Liu, X., Shamloo, M., Malenka, R. C., & Südhof, T. C. (2011) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1111093108 · Cited by 340 · see citing works
Scattoni, M. L., Ricceri, L., & Crawley, J. N. (2010) · Genes Brain & Behavior · doi:10.1111/j.1601-183x.2010.00623.x · Cited by 336 · see citing works
Constraining Heterogeneity: The Social Brain and its Development in Autism Spectrum Disorder
Pelphrey, K., Shultz, S., Hudac, C., & Wyk, B. C. V. (2011) · Journal of Child Psychology and Psychiatry and Allied Disciplines · doi:10.1111/j.1469-7610.2010.02349.x · Cited by 336 · see citing works
Berry-Kravis, E. M., Hessl, D., Rathmell, B., Zarevics, P., Cherubini, M., Walton-Bowen, K., Mu, Y., Nguyen, D. V., Gonzalez-Heydrich, J., Wang, P. P., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3004214 · Cited by 328 · see citing works
Davies, D. M., de Vries, P. J., Johnson, S. R., McCartney, D. L., Cox, J. A., Serra, A. L., Watson, P., Howe, C. J., Doyle, T., Pointon, K. S., et al. (2011) · Clinical Cancer Research · doi:10.1158/1078-0432.ccr-11-0445 · Cited by 305 · see citing works
Betancur, C., Sakurai, T., & Buxbaum, J. D. (2009) · Trends in Neurosciences · doi:10.1016/j.tins.2009.04.003 · Cited by 298 · see citing works
Leigh, M. J. S., Nguyen, D. V., Mu, Y., Winarni, T. I., Schneider, A., Chechi, T., Polussa, J., Doucet, P., Tassone, F., Rivera, S. M., et al. (2013) · Journal of Developmental & Behavioral Pediatrics · doi:10.1097/dbp.0b013e318287cd17 · Cited by 298 · see citing works
Chez, M. G., Chang, M., Krasne, V., Coughlan, C. A., Kominsky, M., & Schwartz, A. (2006) · Epilepsy & Behavior · doi:10.1016/j.yebeh.2005.11.001 · Cited by 275 · see citing works
Angelman syndrome: insights into genomic imprinting and neurodevelopmental phenotypes
Mabb, A. M., Judson, M. C., Zylka, M. J., & Philpot, B. D. (2011) · Trends in Neurosciences · doi:10.1016/j.tins.2011.04.001 · Cited by 263 · see citing works
Toward Fulfilling the Promise of Molecular Medicine in Fragile X Syndrome
Krueger, D. D., & Bear, M. F. (2011) · Annual Review of Medicine · doi:10.1146/annurev-med-061109-134644 · Cited by 259 · see citing works
Silverman, J. L., Smith, D. G., Rizzo, S. J. S., Karras, M. N., Turner, S. M., Tolu, S. S., Bryce, D. K., Smith, D. L., Fonseca, K., Ring, R. H., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003501 · Cited by 251 · see citing works
Henderson, C., Wijetunge, L., Kinoshita, M. N., Shumway, M., Hammond, R. S., Postma, F. R., Brynczka, C., Rush, R., Thomas, A., Paylor, R., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3004218 · Cited by 249 · see citing works
Translational animal models of autism and neurodevelopmental disorders
Crawley, J. N. (2012) · Dialogues in Clinical Neuroscience · doi:10.31887/dcns.2012.14.3/jcrawley · Cited by 227 · see citing works
Open-label add-on treatment trial of minocycline in fragile X syndrome.
Paribello, C., Tao, L., Folino, A., Berry-Kravis, E., Tranfaglia, M., Ethell, I. M., & Ethell, D. W. (2010) · BMC Neurology · doi:10.1186/1471-2377-10-91 · Cited by 223 · see citing works
Open-Label Treatment Trial of Lithium to Target the Underlying Defect in Fragile X Syndrome
Berry-Kravis, E., Sumis, A., Hervey, C., Nelson, M., Porges, S. W., Weng, N., Weiler, I. J., & Greenough, W. T. (2008) · Journal of Developmental & Behavioral Pediatrics · doi:10.1097/dbp.0b013e31817dc447 · Cited by 215 · see citing works
Mines, M. A., Yuskaitis, C. J., King, M. K., Beurel, E., & Jope, R. S. (2010) · PLoS ONE · doi:10.1371/journal.pone.0009706 · Cited by 208 · see citing works
Dabora, S. L., Franz, D. N., Ashwal, S., Sagalowsky, A. I., DiMario, F. J., Miles, D. K., Cutler, D., Krueger, D. A., Uppot, R. N., Rabenou, R. A., et al. (2011) · PLoS ONE · doi:10.1371/journal.pone.0023379 · Cited by 203 · see citing works
Yuskaitis, C. J., Mines, M. A., King, M. K., Sweatt, J. D., Miller, C. A., & Jope, R. S. (2009) · Biochemical Pharmacology · doi:10.1016/j.bcp.2009.09.023 · Cited by 187 · see citing works
Sansone, S. M., Widaman, K. F., Hall, S. S., Reiss, A. L., Lightbody, A. A., Kaufmann, W. E., Berry‐Kravis, E., Lachiewicz, A. M., Brown, E. C., & Hessl, D. R. (2011) · Journal of Autism and Developmental Disorders · doi:10.1007/s10803-011-1370-2 · Cited by 177 · see citing works
Fragile X Syndrome: The GABAergic System and Circuit Dysfunction
Paluszkiewicz, S. M., Martin, B. S., & Huntsman, M. M. (2011) · Developmental Neuroscience · doi:10.1159/000329420 · Cited by 177 · see citing works
Human neuroimaging of oxytocin and vasopressin in social cognition
Zink, C. F., & Meyer-Lindenberg, A. (2012) · Hormones and Behavior · doi:10.1016/j.yhbeh.2012.01.016 · Cited by 176 · see citing works
Regression in Autistic Spectrum Disorders
Stefanatos, G. A. (2008) · Neuropsychology Review · doi:10.1007/s11065-008-9073-y · Cited by 161 · see citing works
Investigating synapse formation and function using human pluripotent stem cell-derived neurons
Kim, J.-E., O'Sullivan, M. L., Sánchez, C. A., Hwang, M., Israel, M. A., Brennand, K., Deerinck, T., Goldstein, L., Gage, F., Ellisman, M., et al. (2011) · Proceedings of the National Academy of Sciences of the United States of America · doi:10.1073/pnas.1007753108 · Cited by 146 · see citing works
Self-citationGenetics of autism spectrum disorders
Kumar, R. A., & Christian, S. L. (2009) · Current Neurology and Neuroscience Reports · doi:10.1007/s11910-009-0029-2 · Cited by 146 · see citing works
Altered ultrasonic vocalizations in a tuberous sclerosis mouse model of autism
Young, D. M., Schenk, A. K., Yang, S., Jan, Y. N., & Jan, L. Y. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1005620107 · Cited by 143 · see citing works
Etherton, M. R., Tabuchi, K., Sharma, M., Ko, J., & Südhof, T. C. (2011) · The EMBO Journal · doi:10.1038/emboj.2011.182 · Cited by 140 · see citing works
Rotschafer, S. E., Trujillo, M. S., Dansie, L. E., Ethell, I. M., & Razak, K. A. (2012) · Brain Research · doi:10.1016/j.brainres.2011.12.041 · Cited by 130 · see citing works
Spooren, W., Lindemann, L., Ghosh, A., & Santarelli, L. (2012) · Trends in Pharmacological Sciences · doi:10.1016/j.tips.2012.09.004 · Cited by 119 · see citing works
Self-citationThe Innovative Medicines Initiative: A European Response to the Innovation Challenge
Goldman, M. (2012) · Clinical Pharmacology & Therapeutics · doi:10.1038/clpt.2011.321 · Cited by 108 · see citing works
Cellular and synaptic network defects in autism
Peça, J., & Feng, G. (2012) · Current Opinion in Neurobiology · doi:10.1016/j.conb.2012.02.015 · Cited by 96 · see citing works
Politte, L. C., & McDougle, C. J. (2013) · Psychopharmacology · doi:10.1007/s00213-013-3068-y · Cited by 96 · see citing works
Roy, S., Watkins, N. W., & Heck, D. H. (2012) · PLoS ONE · doi:10.1371/journal.pone.0044816 · Cited by 88 · see citing works
Detection and Characterization of Copy Number Variation in Autism Spectrum Disorder
Marshall, C. R., & Scherer, S. W. (2011) · Methods in molecular biology · doi:10.1007/978-1-61779-507-7_5 · Cited by 87 · see citing works
Fragile X syndrome—From genes to cognition
Schneider, A., Hagerman, R. J., & Hessl, D. R. (2009) · Developmental Disabilities Research Reviews · doi:10.1002/ddrr.80 · Cited by 87 · see citing works
Reliability of Eye Tracking and Pupillometry Measures in Individuals with Fragile X Syndrome
Farzin, F., Huang, F. S., Hervey, C. M., Berry‐Kravis, E., & Hessl, D. R. (2011) · Journal of Autism and Developmental Disorders · doi:10.1007/s10803-011-1176-2 · Cited by 69 · see citing works
EU-AIMS: a boost to autism research
Murphy, D., & Spooren, W. P. J. M. (2012) · Nature Reviews Drug Discovery · doi:10.1038/nrd3881 · Cited by 66 · see citing works
Innovation in Residency Education and Evolving Pediatric Health Needs
Jones, M. D. (2009) · PEDIATRICS · doi:10.1542/peds.2009-1892 · Cited by 40 · see citing works
Epigenetic Discordance at Imprinting Control Regions in Twins
Ollikainen, M., & Craig, J. M. (2011) · Epigenomics · doi:10.2217/epi.11.18 · Cited by 40 · see citing works
Laverty, H., Gunn, M., & Goldman, M. (2012) · Expert Review of Pharmacoeconomics & Outcomes Research · doi:10.1586/erp.12.59 · Cited by 13 · see citing works
Randomized, Controlled, Phase 2 Trial of STX209 (Arbaclofen) for Social Function in ASD
Delahunty, C. M. (2013) · Cited by 7 · see citing works
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Lord, C., Brugha, T. S., Charman, T., Cusack, J., Dumas, G., Frazier, T., Jones, E. J. H., Jones, R. M., Pickles, A., State, M. W., et al. (2020) · Nature Reviews Disease Primers · doi:10.1038/s41572-019-0138-4 · Cited by 1,859 · see citing works
Colizzi, M., Lasalvia, A., & Ruggeri, M. (2020) · International Journal of Mental Health Systems · doi:10.1186/s13033-020-00356-9 · Cited by 727 · see citing works
Altering the course of schizophrenia: progress and perspectives
Millan, M. J., Andrieux, A., Bartzokis, G., Cadenhead, K., Dazzan, P., Fusar-Poli, P., Gallinat, J., Giedd, J., Grayson, D. R., Heinrichs, M., et al. (2016) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2016.28 · Cited by 516 · see citing works
Identification and validation of biomarkers for autism spectrum disorders
Loth, E., Spooren, W., Ham, L. M., Isaac, M. B., Auriche-Benichou, C., Banaschewski, T., Baron-Cohen, S., Broich, K., Bölte, S., Bourgeron, T., et al. (2016) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2015.7 · Cited by 140 · see citing works
Beversdorf, D. Q., & MISSOURI AUTISM SUMMIT CONSORTIUM* (2016) · Journal of Developmental & Behavioral Pediatrics · doi:10.1097/dbp.0000000000000351 · Cited by 74 · see citing works
Modeling autism-relevant behavioral phenotypes in rats and mice: Do ‘autistic’ rodents exist?
Servadio, M., Vanderschuren, L. J. M. J., & Trezza, V. (2015) · Behavioural Pharmacology · doi:10.1097/fbp.0000000000000163 · Cited by 76 · see citing works
Oxytocin: Parallel Processing in the Social Brain?
Dölen, G. (2015) · Journal of Neuroendocrinology · doi:10.1111/jne.12284 · Cited by 65 · see citing works
Aligning physiology with psychology: Translational neuroscience in neuropsychiatric drug discovery
McArthur, R. A. (2017) · Neuroscience & Biobehavioral Reviews · doi:10.1016/j.neubiorev.2017.02.004 · Cited by 42 · see citing works
Pharmacogenomic Medicine in Autism: Challenges and Opportunities
Bowers, K., Lin, P.-I., & Erickson, C. (2015) · Pediatric Drugs · doi:10.1007/s40272-014-0106-0 · Cited by 22 · see citing works
- Authors
- Ghosh, A., Michalon, A., Lindemann, L., Fontoura, P., & Santarelli, L.
- Affiliations
- F. Hoffmann-La Roche AG (CH)
- Corresponding author
- Ghosh, Anirvan
- Venue
- Nature Reviews Drug Discovery
- Published
- 2013-10
- Volume
- 12
- Issue
- 10
- Pages
- 777-790
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- OpenAlex
- W1973491179
- Cited by
- 108
- References
- 87
- Citations by year
- 2026: 1 · 2025: 7 · 2024: 6 · 2023: 6 · 2022: 10 · 2021: 6 · 2020: 6 · 2019: 4 · 2018: 6 · 2017: 11 · 2016: 14 · 2015: 20 · 2014: 10 · 2013: 1
- FWCI
- 1.29
- Type
- journal article
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Reviews Drug Discovery (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Simons Foundation
- Autism Research Foundation
- European Federation of Pharmaceutical Industries and Associations
- Topics
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Keywords
- autism spectrum disorder, drug discovery, fragile X syndrome, tuberous sclerosis complex, single-gene mutations, animal models of autism, induced pluripotent stem cells, drug screening, disease modification, clinical trials, target identification, drug profiling, academia-industry collaboration
- MeSH terms
- Animals, Child, Child Development Disorders, Pervasive, Clinical Trials as Topic, Cooperative Behavior, Disease Models, Animal, Drug Industry, Humans, Drug Design, Drug Discovery, Molecular Targeted Therapy
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