Autism spectrum disorder (ASD) refers to a heterogeneous group of etiologically and phenotypically complex neurodevelopmental disorders that affects as many as 1 in 68 children and is the fastest-growing serious developmental disability in the United States. Marked and sustained problems in social interaction and restricted interests/repetitive behavior are the essential defining features. These core symptoms are often comorbid with intellectual disability, epilepsy, attention-deficit hyperactivity disorder, sleep disorders, anxiety, and other disorders. Furthermore, as implied by the word “spectrum,” ASD phenotypes range from mild behavioral and personality traits to severe and debilitating impairments.There is currently no medical cure, single diagnostic test, or biological marker for ASD. The only two drugs approved for the medical “treatment” of autism are risperidone and aripiprazole, both of which are atypical antipsychotics intended for the treatment of irritability associated with ASD. To this date, the U.S. Food and Drug Administration has yet to approve a single drug for the treatment of autism’s core symptoms, likely owing to our nebulous understanding of the underlying neurobiology of ASD. However, there are several highly effective, evidence-based behavioral therapies in practice. These include a range of evidence-based treatments that include Applied Behavior Analysis (ABA), developmentally based interventions, hybrid models (combining aspects of ABA and developmental approaches), and “eclectic” models. Given the prevalence of ASD and the time and labor-intensive nature of such treatment paradigms, an effective pharmacologic agent for treating ASD’s core symptoms is a great unmet need.In this focus issue on Autism Spectrum Disorders, we have selected articles that highlight the interdisciplinary nature of the study of autism pathogenesis and treatment, including three reviews. The first, by Ardhanareeswaran et al., describes our current understanding of the underlying neurobiology of ASD and how stem cells — specifically, induced pluripotent stem cells (iPSCs) — might advance that understanding. They also detail the application of omics approaches and genome editing technologies to the iPSC system.Prior research in social cognition has focused on studying the social brain in isolation. Since ASD is a disorder characterized by core deficits in social interaction, reciprocity, and communication, it is critical to study live interactions in order to better understand the neural signatures of ASD. Rolison et al. provide an overview of interactive social neuroscience experiments involving the simultaneous recording of two or more brains in the context of ASD.Since there is currently no straightforward diagnostic tool available for ASD, several groups have recently focused efforts on finding biomarkers. Port et al. summarize the emerging field of biomarkers in ASD. They describe several potential biomarkers, including middle latency delays (M50/100), mismatched negativity latency, and Gamma-band activity. Additionally, they investigate the relation of these biomarkers to symptomology, core domains of dysfunction (e.g., language impairment), and putative neurobiological underpinnings.This issue also includes three original research articles, a perspective, and a mini review that each highlight unique areas of interest in autism diagnosis and treatment. Ventola et al. describe the response of two preschool-aged children to Pivotal Response Treatment, which is derived from Applied Behavior Analysis, while Gelbar et al. present the results of a survey of ASD patients in the context of higher education. Grapel et al. discuss the usefulness of sensory criteria in ASD diagnosis. Klin et al., in their perspective, describe the unique challenges that military families with autistic children face. They argue for the implementation of evidence-based solutions that focus on reducing the age of diagnosis, improving access to early intervention, and personalizing services for these children and their families. Finally, in a mini review of gender issues in ASD, van Schalkwyk et al. discuss gender identity disorder (GID) and gender dysphoria (GD) in the context of ASD, arguing for the facilitation of larger conversations on gender in identity formation by clinicians with their ASD patients.Together, we hope these articles give a glimpse of the many facets that make up ASD research from basic science to translational science to health care policy. All three are absolutely vital to making a difference in the lives of patients with ASD. We also hope these papers, in describing the current state of their respective subfields, highlight areas of unmet needs and potential approaches for future research investigators to pursue. In a time of growing disease burden and controversy on diagnostic methodology, it is crucial that we elucidate the biological abnormalities that give rise to ASD, while simultaneously providing accessible, rigorously validated behavioral treatments and services to current ASD patients and their families.