Bourgeron Thomas
a Thomas Bourgeron, Human Genetics and Cognitive Functions Unit, Institut Pasteur, Paris, France, CNRS UMR3571 Genes, Synapses and Cognition, Institut Pasteur, Paris, France, Human Genetics and Cognitive Functions, University Paris Diderot, Sorbonne Paris Cité, Paris, France, Fondation FondaMental, Créteil, France, Gillberg Neuropsychiatry Centre, Sahlgrenska Academy, University of Gothenburg , Gothenburg , Sweden.
Nord J Psychiatry. 2016;70(1):1-9. doi: 10.3109/08039488.2015.1042519. Epub 2015 May 14.
ESSENCE refers to early symptomatic syndromes eliciting neurodevelopmental clinical examinations. It includes a broad range of early onset neurodevelopmental disorders affecting more than 10% of children before 5 years of age. ESSENCE includes among others attention deficit hyperactivity disorder (ADHD), intellectual disability (ID) and autism spectrum disorders (ASD). Some degree of disability is the rule rather than the exception. The causes are heterogeneous ranging from extreme social deprivation, pre- and perinatal risk factors, genetic and metabolic diseases, immune and infectious disorders, nutritional factors, physical trauma, and postnatal toxic and environmental factors (and combinations/interactions of some or several of these). Treatments often involve a combination of psychoeducational interventions, home- and school-based programmes, and medication. Here, I will first briefly review our main knowledge on the biological pathways associated with early onset neurodevelopmental disorders and will provide useful links to be informed of the progress in the field. Five main pathways are associated with ASD and ID: chromatin remodelling, cytoskeleton dynamics, mRNA translation, metabolism and synapse formation/function. I will then detail three propositions coming from institutions, researchers and/or communities of patients and families to foster research: 1) to use more dimensional and quantitative data than diagnostic categories; 2) to increase data sharing and research on genetic and brain diversity in human populations; 3) to involve patients and relatives as participants for research. Finally, I will provide examples of very stimulating initiatives towards a more inclusive world for individuals with ESSENCE.
ESSENCE指引发神经发育临床检查的早期症状综合征。它包括一系列广泛的早发性神经发育障碍,影响超过10%的5岁前儿童。ESSENCE尤其包括注意力缺陷多动障碍(ADHD)、智力残疾(ID)和自闭症谱系障碍(ASD)。某种程度的残疾是常态而非例外。其病因多种多样,包括极端的社会剥夺、产前和围产期风险因素、遗传和代谢疾病、免疫和感染性疾病、营养因素、身体创伤以及产后有毒和环境因素(以及其中一些或几种因素的组合/相互作用)。治疗通常涉及心理教育干预、家庭和学校项目以及药物治疗的组合。在此,我将首先简要回顾我们对与早发性神经发育障碍相关的生物学途径的主要认识,并提供有用的链接以了解该领域的进展。有五条主要途径与ASD和ID相关:染色质重塑、细胞骨架动力学、mRNA翻译、代谢以及突触形成/功能。然后,我将详细阐述来自机构、研究人员和/或患者及家庭群体提出的三项促进研究的建议:1)使用比诊断类别更多的维度和定量数据;2)增加人类群体中基因和大脑多样性的数据共享和研究;3)让患者及其亲属作为研究参与者。最后,我将举例说明为患有ESSENCE的个体创造一个更具包容性的世界的非常鼓舞人心的举措。