Department of Neurobiology and Anatomy, Drexel University College of Medicine, Philadelphia, PA 19129, USA.
Genes (Basel). 2024 Sep 17;15(9):1214. doi: 10.3390/genes15091214.
The genetic basis of schizophrenia (SZ) remains elusive despite its characterization as a highly heritable disorder. This incomplete understanding has led to stagnation in therapeutics and treatment, leaving many suffering with insufficient relief from symptoms. However, recent large-cohort genome- and exome-wide association studies have provided insights into the underlying genetic machinery. The scale of these studies allows for the identification of ultra-rare mutations that confer substantial disease risk, guiding clinicians and researchers toward general classes of genes that are central to SZ etiology. One such large-scale collaboration effort by the Schizophrenia Exome Sequencing Meta-Analysis consortium identified ten, high-risk, ultra-rare, protein-truncating variants, providing the clearest picture to date of the dysfunctional gene products that substantially increase risk for SZ. While genetic studies of SZ provide valuable information regarding "what" genes are linked with the disorder, it is an open question as to "when" during brain development these genetic mutations impose deleterious effects. To shed light on this unresolved aspect of SZ etiology, we queried the BrainSpan developmental mRNA expression database for these ten high-risk genes and discovered three general expression trajectories throughout pre- and postnatal brain development. The elusiveness of SZ etiology, we infer, is not only borne out of the genetic heterogeneity across clinical cases, but also in our incomplete understanding of how genetic mutations perturb neurodevelopment during multiple critical periods. We contextualize this notion within the National Institute of Mental Health's Research Domain Criteria framework and emphasize the utility of considering both genetic variables and developmental context in future studies.
尽管精神分裂症 (SZ) 被认为是一种高度遗传性疾病,但其遗传基础仍难以捉摸。这种不完全的理解导致治疗和治疗停滞不前,许多患者的症状得不到充分缓解。然而,最近的大规模基因组和外显子组全基因组关联研究为深入了解潜在的遗传机制提供了线索。这些研究的规模允许识别出赋予大量疾病风险的超罕见突变,指导临床医生和研究人员关注对 SZ 病因学至关重要的一般基因类别。Schizophrenia Exome Sequencing Meta-Analysis 联盟的一项此类大规模合作努力确定了十个高风险、超罕见、蛋白截断的变异体,为目前最清晰地描绘出功能失调的基因产物提供了依据,这些基因产物极大地增加了 SZ 的风险。虽然 SZ 的遗传研究提供了有关与该疾病相关的“哪些”基因的宝贵信息,但这些基因变异在大脑发育的“何时”对产生有害影响仍然是一个悬而未决的问题。为了阐明 SZ 病因学中这个未解决的方面,我们在 BrainSpan 发育性 mRNA 表达数据库中查询了这十个高风险基因,并在产前和产后大脑发育过程中发现了三个总体表达轨迹。我们推断,SZ 病因的难以捉摸不仅源于临床病例中的遗传异质性,还源于我们对遗传突变如何在多个关键时期干扰神经发育的理解不完整。我们将这个概念置于国家心理健康研究所的研究领域标准框架内,并强调在未来的研究中考虑遗传变量和发育背景的实用性。