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BACKGROUND AND PURPOSE: Tay-Sachs disease is a rare and often fatal, autosomal recessive, lysosomal storage disease. Deficiency in ß-hexosaminidase leads to accumulation of GM2 ganglioside resulting in neuronal swelling and degeneration. Typical onset is in infancy with developmental regression and early death. Late-onset Tay-Sachs disease (LOTS) is extremely rare, especially in the non-Ashkenazi Jewish population, and is characterized by a more indolent presentation typically encompassing features of cerebellar and anterior horn cell dysfunction in addition to extrapyramidal and neuropsychiatric symptoms. CASES: A case series of four unrelated patients of non-Ashkenazi Jewish origin with a predominantly, and in some cases pure, neuromuscular phenotype with evidence of a motor neuronopathy on electromyography is presented. Cerebellar atrophy, reported to be a ubiquitous feature in LOTS, was absent in all patients. CONCLUSION: This case series provides evidence to support a pure neuromuscular phenotype in LOTS, which should be considered in the differential diagnosis of anterior horn cell disorders.
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Transtornos Mentais , Doença de Tay-Sachs , Humanos , Doença de Tay-Sachs/diagnóstico , Doença de Tay-Sachs/genética , Doença de Tay-Sachs/psicologia , Fenótipo , CerebeloRESUMO
AIM: To examine child-led goal setting and evaluation tools and approaches for children with a disability or developmental delay. METHOD: Six databases were searched for studies that included population (children aged less than 18 years with disability or developmental delay); construct (child-led goal setting tool or approach); and context (developmental therapy or rehabilitation). The utility of tools and approaches across the goal setting and evaluation process was investigated using abductive content analysis. RESULTS: Fifty articles met the inclusion criteria. Three approaches and four tools for child-led goal setting and evaluation were identified. No studies reported the clinimetric properties of tools specifically for child self-respondents. Qualitative analysis revealed six distinct goal phases in which tools and approaches were used, which were synthesized into a new framework for child-led goal setting and evaluation titled DECIDE: Direct children to goal setting; Elicit goal topics and priorities; Construct a goal statement; Indicate baseline goal performance; Develop an action plan to address the goal; and Evaluate goal progress after the intervention. INTERPRETATION: Children actively participated in goal setting and evaluation across six DECIDE goal phases. Further clinimetric information is required to support use of goal setting and evaluation tools with child self-respondents. Future research should emphasize the development of multi-phase goal setting tools and approaches for diverse populations of children.
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A 72-year-old woman had a warty growth 'burnt off' with a herbal remedy by a local farmer in rural Ireland. We saw the patient a number of years later in clinic and she brought the specimen with her, which was processed by the pathology department. We report her case and some interesting background information regarding escharotic agents.
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Verrugas/terapia , Idoso , Feminino , Medicina Herbária , Humanos , IrlandaRESUMO
Motor neuron disease (MND) is a neurodegenerative disorder which leads to progressive muscle weakness including respiratory muscle decline. The introduction of non-invasive ventilation (NIV) has been shown to improve quality of life, survival and slow the rate of pulmonary function decline. A retrospective chart analysis of patients who attended the MND clinic from 2014 to 2019 at a tertiary-referral, academic, teaching hospital was carried out to evaluate if NIV and greater compliance with NIV was associated with improved survival. 111 patients were included. The mean age at diagnosis was 63.8 years and 61.3% were males. 66.7% of our cohort used NIV and of this 66.7%, 44.1% were compliant. There was a significantly longer survival in those who used NIV (p = 0.002) and in those who used NIV optimally (p = 0.02) when both groups were compared to those who did not use NIV. In the bulbar MND group those who were compliant with NIV survived longer than who those who did not use NIV (p = 0.001). We found a significantly longer survival with the use of NIV, the use of NIV optimally and with use of NIV in those with bulbar onset MND compared to those who did not use NIV.
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Doença dos Neurônios Motores , Ventilação não Invasiva , Insuficiência Respiratória , Estudos de Coortes , Humanos , Masculino , Doença dos Neurônios Motores/terapia , Qualidade de Vida , Insuficiência Respiratória/terapia , Estudos RetrospectivosRESUMO
GANT61-D is an important hedgehog pathway inhibitor and an interesting ligand candidate for metal coordination. The first examples of metal complexes of the potent hedgehog pathway inhibitor GANT61-D are described. The reaction of Ni(II), Pd(II), and Pt(II) precursors with the hedgehog pathway inhibitor GANT61-D gave [NiII(GANT61-D)(OH2)3(µ2-SO4)(µ3-SO4)] (1), [PdII(Cl)(GANT61-D)]Cl (2), [PtII(Cl)(GANT61-D)]Cl, and [PtII(CBDCA-2H)(GANT61-D)]. X-ray crystal structure analysis revealed that GANT61-D is a versatile N-donor ligand that can act as a bidentate ligand via the diaminopropane (DAP) N atoms or a tridentate ligand via the DAP N atoms and one dimethylaniline N atom. Protonation constants of the GANT61-D ligand in water and in a 60:40 (w/w) dimethyl sulfoxide-water solvent mixture were determined. Potentiometric and spectroscopic data on the NiII(GANT61-D) system indicate the formation of octahedral 1:1 species with medium stability in solution. 1 and 2 exhibited noteworthy in vitro cytotoxicity against medulloblastoma cancer cells.
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Antineoplásicos/farmacologia , Complexos de Coordenação/farmacologia , Níquel/farmacologia , Paládio/farmacologia , Platina/farmacologia , Antineoplásicos/síntese química , Antineoplásicos/química , Linhagem Celular Tumoral , Proliferação de Células/efeitos dos fármacos , Complexos de Coordenação/síntese química , Complexos de Coordenação/química , Cristalografia por Raios X , Ensaios de Seleção de Medicamentos Antitumorais , Humanos , Modelos Moleculares , Estrutura Molecular , Níquel/química , Paládio/química , Platina/químicaRESUMO
BACKGROUND: People with schizophrenia are ten times more likely to commit homicide than a member of the general population. The relationship between symptoms of schizophrenia and acts of violence is unclear. There has also been limited research on what determines the seriousness and form of violence, such as reactive or instrumental violence. Moral cognition may play a paradoxical role in acts of violence for people with schizophrenia. Thoughts which have moral content arising from psychotic symptoms may be a cause of serious violence. METHOD: We investigated if psychotic symptoms and moral cognitions at the time of a violent act were associated with acts of violence using a cross-sectional national forensic cohort (n = 55). We examined whether moral cognitions were associated with violence when controlling for neurocognition and violence proneness. We explored the association between all psychotic symptoms present at the time of the violent act, psychotic symptoms judged relevant to the violent act and moral cognitions present at that time. Using mediation analysis, we examined whether moral cognitions were the missing link between symptoms and the relevance of symptoms for violence. We also investigated if specific moral cognitions mediated the relationship between specific psychotic symptoms, the seriousness of violence (including homicide), and the form of violence. RESULTS: Psychotic symptoms generally were not associated with the seriousness or form of violence. However, specific moral cognitions were associated with the seriousness and form of violence even when controlling for neurocognition and violence proneness. Specific moral cognitions were associated with specific psychotic symptoms present and relevant to violence. Moral cognitions mediated the relationship between the presence of specific psychotic symptoms and their relevance for violence, homicide, seriousness of violence, and the form of violence. CONCLUSIONS: Moral cognitions including the need to reduce suffering, responding to an act of injustice or betrayal, the desire to comply with authority, or the wish to punish impure or disgusting behaviour, may be a key mediator explaining the relationship between psychotic symptoms and acts of violence. Our findings may have important implications for risk assessment, treatment and violence prevention.
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Cognição , Princípios Morais , Transtornos Psicóticos/psicologia , Violência/psicologia , Adulto , Estudos de Coortes , Estudos Transversais , Feminino , Homicídio/psicologia , Humanos , Masculino , Pessoa de Meia-IdadeRESUMO
The objective was to determine plasma levels of pro- (IL-12p70/IL-6) and anti-inflammatory (IL-10) cytokines before and after cycle ergometer training in healthy control (HC) and people with multiple sclerosis (pwMS), and to correlate plasma cytokines with physical/mental health. Study participants cycled for 30 min at 65-75% age-predicted maximal heart rate, twice a week for 8 weeks during supervised sessions. We determined that plasma IL-10 expression was lower in pwMS, compared to HCs, and that exercise augmented IL-10 in pwMS to baseline levels in HCs. Furthermore, plasma isolated from pwMS displayed enhanced expression of the pro-inflammatory cytokines IL-12p70/IL-6. Plasma cytokine signatures correlated with physical/mental health. Overall, this study highlights the potential of a short-term exercise programme to regulate circulating cytokine profiles with relevance to pwMS.
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Ciclismo , Terapia por Exercício/métodos , Interleucina-10/sangue , Esclerose Múltipla/sangue , Esclerose Múltipla/terapia , Adulto , Ciclismo/fisiologia , Ergometria , Humanos , Subunidade p35 da Interleucina-12/sangue , Interleucina-6/sangueRESUMO
Following publication the authors informed the Journal that the published version of this article contained a mistake. All occurrences of pg/µl found in the original article should be changed to pg/L. The original article has been corrected. The correction has no impact on the conclusions drawn in the manuscript.
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OBJECTIVES: This study explored how signs and symptoms of pyruvate kinase (PK) deficiency, a rare hemolytic anemia caused by mutations in the PKLR gene, impacts patients' health-related quality of life (HRQoL). METHODS: Interviews with 21 adults with PK deficiency in the United States, Netherlands, and Germany were conducted. Participants were asked to describe signs, symptoms, and impacts of the disease on their daily lives. Interviews were transcribed and analyzed using qualitative analysis methods. RESULTS: The most common signs and symptoms reported were yellow eyes (n = 19), tiredness (n = 18), yellow skin (n = 17), fatigue (n = 15), low energy (n = 13), and shortness of breath (n = 13). Furthermore, signs and symptoms of PK deficiency negatively impact the ability to perform physical activities, appearance, social activities, emotional states, activities of daily living, leisure activities, work and/or school, sleep, and cognitive states of those living with PK deficiency. A conceptual model is presented that further demonstrates the profound impact that signs and symptoms of PK deficiency have on dimensions of patients' HRQoL. CONCLUSIONS: This is the first study that provides patient perspective on the burden of living with PK deficiency and lays the foundation for future studies to examine the effect of pharmacologic interventions on overall HRQoL for patients living with PK deficiency.
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Anemia Hemolítica Congênita não Esferocítica/epidemiologia , Efeitos Psicossociais da Doença , Piruvato Quinase/deficiência , Erros Inatos do Metabolismo dos Piruvatos/epidemiologia , Atividades Cotidianas , Anemia Hemolítica Congênita não Esferocítica/diagnóstico , Anemia Hemolítica Congênita não Esferocítica/psicologia , Suscetibilidade a Doenças , Emoções , Humanos , Percepção , Erros Inatos do Metabolismo dos Piruvatos/diagnóstico , Erros Inatos do Metabolismo dos Piruvatos/psicologia , Qualidade de Vida , Autoimagem , Avaliação de SintomasRESUMO
In this pilot study, we investigate whether a routine cycle ergometry training programme has therapeutic potential in individuals with multiple sclerosis (MS) by improving quality of life (QOL) and depressive symptomatology, while ameliorating cognitive disturbances. Healthy volunteers and MS patients cycled for 30 min at 65-75% age-predicted maximal heart rate on a recumbent ergometer, with this session repeated twice a week for 8 weeks. QOL, depressive symptomatology and cognitive function were assessed pre- and post-exercise using the MS Quality of Life-54 (MSQOL-54) questionnaire, 16-item Quick Inventory of Depressive Symptomatology (QIDS-SR16) questionnaire and the Cambridge Neuropsychological Test Automated Battery (CANTAB), respectively. We determined that QOL was lower in MS patients, compared to healthy subjects, with a reduction in physical and mental health summary scores observed. Exercise improved both physical and mental health scores in MS patients. In support of this, exercise was shown to reduce depressive symptomatology in MS patients. Exercise was also associated with an improvement in visual sustained attention, executive function/cognitive flexibility and hippocampal-dependent visuospatial memory in patients. Overall, this study identifies a short-term exercise programme that improves physical and mental health, while reducing depressive symptomatology and cognitive dysfunction in MS.
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Cognição , Depressão/terapia , Terapia por Exercício , Esclerose Múltipla/psicologia , Esclerose Múltipla/terapia , Qualidade de Vida , Adulto , Índice de Massa Corporal , Peso Corporal , Depressão/fisiopatologia , Exercício Físico/psicologia , Terapia por Exercício/instrumentação , Feminino , Humanos , Fatores Imunológicos/uso terapêutico , Masculino , Esclerose Múltipla/fisiopatologia , Testes Neuropsicológicos , Aptidão Física , Projetos Piloto , Resultado do TratamentoRESUMO
BACKGROUND: Neuropathic pain (NP) is a complex, chronic pain state initiated by a primary lesion or dysfunction of the nervous system and presents as a variety of symptoms across multiple disease states. OBJECTIVE: To develop a patient-centred conceptual model of symptoms and impacts in subjects with diabetic peripheral neuropathy (DPN) or post-herpetic neuralgia (PHN) that can inform the measurement strategy in clinical trials. METHOD: Thirty subjects with DPN or PHN participated in in-person interviews which were performed until saturation was achieved. Transcripts were analysed in ATLAS.ti. RESULTS: Interviews were completed with DPN subjects (United States, n = 10; Japan, n = 10) and PHN subjects (United States, n = 5; Japan, n = 5). Numbness and tingling were frequently reported symptoms in the DPN population while itchiness and hypersensitivity were predominant in PHN. Both populations experienced burning and ache/soreness with similar frequency. DPN subjects experienced pain primarily in their lower extremity (eg feet, ankles), while PHN subjects experienced pain primarily in the chest and back. Impacts reported by DPN subjects included difficulty walking, sleep disturbance and climbing stairs. Impacts in PHN subjects included sleep disturbance, avoidance of physical contact, being angry/frustrated and being sad/depressed. Overall, concepts in Japan were not qualitatively different from the United States. Conceptual models of NP were generated based on the concepts elicited. CONCLUSIONS: This research highlights core concepts to measure from the patient's perspective. Moreover, it enables the assessment of existing measures, the possible modification of these measures, or if a new NP measure with improved sensitivity and responsiveness is merited.
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Dor Crônica , Neuropatias Diabéticas/complicações , Hipestesia , Neuralgia Pós-Herpética/complicações , Adulto , Idoso , Povo Asiático/estatística & dados numéricos , Feminino , Humanos , Entrevistas como Assunto , Japão , Masculino , Pessoa de Meia-Idade , Pesquisa Qualitativa , Qualidade de Vida , Estados Unidos , População Branca/estatística & dados numéricosRESUMO
BACKGROUND: There have been no previous population-based studies of adult neuromuscular disease (NMD) in the Republic of Ireland (RoI). This article describes methods and case-ascertainment strategies used to identify patients with inherited and acquired NMD for the purpose of obtaining the prevalence of these disorders in the RoI. METHODS: This epidemiological study was conducted between January 2012 and January 2014. Prospective and retrospective (until 1990) case identification of adults with inherited and acquired NMD have been carried out. Multiple countrywide patient identification sources including neuromuscular clinics, hospital neurology databases, the hospital in-patient enquiry (HIPE) system of each hospital and the records of Muscular Dystrophy Ireland, a nonprofit organisation, were used. RESULTS: In total, 3,724 potential cases were identified. Of these, 1,083 were excluded because 869 cases represented duplicates or triplicates, 133 were coded incorrectly in HIPE, 74 patients were deceased and 7 patients had moved out of the country. The highest number of cases was identified in neurology databases and HIPE (1,724 and 884, respectively). A total of 2,641 individuals fulfilled the inclusion criteria and were included in the study. CONCLUSION: Detailed epidemiological data of this nature is difficult to acquire in the current structure of the Irish health service, requiring multiple sources including input from voluntary patient organisations. The development of a national patient registry for some or all of these conditions would greatly facilitate standardised data recording, giving a true picture of the burden of neuromuscular diseases in a population.
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Projetos de Pesquisa Epidemiológica , Doenças Neuromusculares/epidemiologia , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Feminino , Humanos , Irlanda/epidemiologia , Masculino , Pessoa de Meia-Idade , Doenças Neuromusculares/diagnóstico , Prevalência , Estudos Prospectivos , Estudos Retrospectivos , Adulto JovemRESUMO
BACKGROUND: A call to action is in effect for nurses to be change agents and bridge the gaps between the delivery of health care and the social needs of individuals, families, and communities. Response to this charge requires nurses to address long-standing inequity in health care policy and practice realms. METHOD: This article describes the creative teaching-learning approach of critical reflection practice as a first step in developing skills and attitude for nurses to do this work. RESULTS: Early observations of the effect of critical reflection practice on learners show improved appreciation for the intersection of social, economic, and political dimensions operating in health care policy and practice that influence health inequities. CONCLUSION: When nurses engage in a practice of critical reflection, they are more likely to identify offensive social determinants, act to ameliorate disparities, and advance the agenda for health equity. [J Nurs Educ. 2023;62(5):312-315.].
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Educação em Enfermagem , Equidade em Saúde , Humanos , Atenção à Saúde , Aprendizagem , Política de SaúdeRESUMO
INTRODUCTION: Hereditary amyloidogenic transthyretin (ATTR) amyloidosis is an autosomal dominant, multi-systemic and progressive disorder characterised by polyneuropathy, cardiomyopathy and dysautonomia to varying degrees. In Ireland, the p.Thr80Ala mutation has been well documented, but little has been reported about a second variant, the p.His110Asp mutation first discovered in a family native to county Cork. Here we elaborate on the phenotype of this recently identified mutation using an extended pedigree of the original kindred and include for the first time a second affected family. MATERIALS AND METHODS: Patients attending our centre with confirmed or suspected ATTR amyloidosis as a result of a p.His110Asp mutation were identified. Detailed chart reviews and patient interviews were completed. Data on symptoms, examination findings, neurophysiology, histology, biochemistry, and cardiac investigations were gathered. A large extended pedigree was plotted. RESULTS: A total of 17 members across four generations of one kindred, and 2 members of a previously unreported family were identified. A phenotype of progressive late-onset polyneuropathy with cardiac involvement was common to both families. An early manifestation was carpal tunnel syndrome, preceededing neuropathy by many years. Gastrointestinal and urinary symptoms were common. DISCUSSION: We describe a wider phenotype of the p.His110Asp mutation of transthyretin in two Irish families. Importantly, we describe cardiac involvement which was not previously emphasised. The discovery of a new unrelated family highlights the importance of clinical suspicion even in those without known family history. We suggest that this second important transthyretin mutation should be considered in patients of Irish origin.
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Neuropatias Amiloides Familiares , Polineuropatias , Neuropatias Amiloides Familiares/diagnóstico , Neuropatias Amiloides Familiares/genética , Humanos , Mutação/genética , Fenótipo , Pré-Albumina/genéticaRESUMO
The first example of a Pt complex of GANT61, a hedgehog (Hh) pathway inhibitor is reported. Reaction of cis-[Pt(II)Cl2(dmso)2] with one equivalent of 4-pyridine carboxaldehyde (4-PCA, control ligand) or one equivalent of GANT61 (Hh pathway inhibitor) in acetone at rt for 30 minutes afforded trans-[Pt(II)Cl2(dmso)(4-PCA)] (1) and trans-[Pt(II)Cl2(dmso)(GANT61)] (2) respectively, where 4-PCA and GANT61 are N-donor ligands. The structures of 1 and 2 were fully characterised by elemental analysis, 1H NMR, 13C NMR and IR spectroscopy and X-ray crystallography. 1 and 2 undergo isomerisation from trans- to cis-in solution and therefore the biological activity of 2 is also associated with the cis-configuration. The in vitro cytotoxicity data show that 2 is a potent inhibitor of the growth of breast CSC-depleted HMLER and breast CSC-enriched HMLER-shEcad cells. Furthermore 2 markedly reduced the size and viability and significantly reduced the number of CSC-enriched HMLER-shEcad mammospheres formed. 2 also induced apoptosis with low micromolar IC50 values against two triple negative breast cancer lines, MDA-MB-231 (MDA231) and BT549. 2, which possesses the Hh pathway inhibitor GANT61 as an N donor ligand exhibits far superior anti-CSC activity including in the CSC-enriched mammosphere model and activity against TNBC cells as compared to its control analogue, the trans-Pt(II) 4-PCA complex 1. The trans-Pt GANT61 complex 2 has also been shown to cause DNA damage and inhibit the Hh pathway at the level of GLI.
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Neoplasias de Mama Triplo Negativas , Humanos , Neoplasias de Mama Triplo Negativas/tratamento farmacológico , Proteínas Hedgehog , Ligantes , Células-Tronco NeoplásicasRESUMO
BACKGROUND: Late-onset Tay-Sachs disease (LOTS) is an autosomal-recessive lysosomal storage disease caused by deficient ß-hexosaminidase A activity. LOTS is rare in the Ashkenazi Jews, but even rarer in the non-Jewish population. CASES: We report an Irish family expanding the LOTS phenotype (ataxia, diffuse muscle wasting, dystonia, chorea, belly dancer's dyskinesia, and neuropsychiatric features) associated with the known HEXA variant 1073 + 1G > A and a novel variant c.459 + 24G > C. CONCLUSIONS: LOTS should be considered in patients with similar symptoms and cerebellar atrophy on brain imaging.
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Historically, electromyography (EMG) is utilized early in the diagnostic evaluation of neuromuscular disorders, but its importance may be diminishing with more sophisticated genetic, imaging and immunohistochemistry investigations now available. In the present study, the diagnostic yield of EMG at predicting pathological abnormalities confirmed by muscle biopsy was determined at our neuroscience center. A retrospective study of consecutive cases reviewed at neuromuscular multidisciplinary meetings between 2007 and 2016 identified patients who had EMG and muscle biopsy as part of their diagnostic evaluation. EMG and biopsy findings were categorized as myopathic, neurogenic or normal. The diagnostic accuracy was determined by calculating the concordance between EMG and pathological findings. Of the 175 cases included in the analysis, there was definite concordance between EMG and muscle biopsy findings in 134 cases (76.6%). Abnormal EMG produced sensitivity of 87% and specificity of 65% for abnormal muscle biopsy. Seventeen patients had a normal EMG and an abnormal muscle biopsy, of which 6 had histopathological findings consistent with mitochondrial myopathy, central core myopathy or glycogen storage disorder. Conflicting EMG and muscle biopsy findings were observed in 10 cases. Inclusion body myositis, chronic neuromuscular disorders and dual pathologies were associated with discordant findings. This study demonstrates that EMG has accurate predictive value in diagnosing neuromuscular disorders at our neuroscience center. EMG retains a vital role, particularly in initial diagnostic evaluations of neuromuscular disorders.
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Doenças Neuromusculares , Biópsia , Eletromiografia , Humanos , Músculos , Doenças Neuromusculares/diagnóstico , Estudos RetrospectivosRESUMO
BACKGROUND AND OBJECTIVES: Research indicates the value of targeting emotional regulation (ER) skills in psychological interventions for psychosis. These skills can be delivered in a group format, thereby increasing access to therapy. This pilot study examined the acceptability and clinical effects of teaching ER skills in The Living Through Psychosis (LTP) group programme. METHODS: Patients with a psychotic illness were offered the LTP programme, comprising eight sessions over four weeks. Measures were completed by 55 participants. Acceptability was assessed by attendance rates and group cohesion. Measures of intervention targets, recovery and clinical outcomes were completed at baseline, pre-group, post-group, and one-month follow-up. RESULTS: High group attendance and cohesion support the acceptability of the group. Participants reported less difficulty with ER (Coeff. = -8.29, 95% CI: -13.40 to -3.18, within participant uncontrolled effect size (ES) d = 0.29), increased mindful relating to distressing symptoms (Coeff. = 11.20, 95% CI: 7.02 to 15.38, d = 0.65), and improvements in recovery dimensions (Coeff. = 10.07, 95% CI: 5.6 to 14.54, d = 0.42) from pre-to post-intervention, and maintained at one-month follow-up. Participants' hallucinations and delusions reduced from pre-intervention to follow-up (t(18) = 4.64, p < 0.001; t(18) = 5.34, p < 0.001). There was no change in fear of relapse. LIMITATIONS: The uncontrolled, pre-post design precluded blinded assessments, and may have inflated effect sizes. Other factors may have contributed to the improvements. CONCLUSIONS: The LTP programme was acceptable to people with psychosis. The preliminary findings indicate the potential utility of teaching ER and mindfulness skills in a brief group programme. Findings require replication in a randomized controlled trial.
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Regulação Emocional , Atenção Plena , Transtornos Psicóticos , Alucinações , Humanos , Projetos Piloto , Transtornos Psicóticos/terapiaRESUMO
The SARS-CoV-2 pandemic has affected more than 185 million people worldwide resulting in over 4 million deaths. To contain the pandemic, there is a continued need for safe vaccines that provide durable protection at low and scalable doses and can be deployed easily. Here, AAVCOVID-1, an adeno-associated viral (AAV), spike-gene-based vaccine candidate demonstrates potent immunogenicity in mouse and non-human primates following a single injection and confers complete protection from SARS-CoV-2 challenge in macaques. Peak neutralizing antibody titers are sustained at 1 year and complemented by functional memory T cell responses. The AAVCOVID vector has no relevant pre-existing immunity in humans and does not elicit cross-reactivity to common AAVs used in gene therapy. Vector genome persistence and expression wanes following injection. The single low-dose requirement, high-yield manufacturability, and 1-month stability for storage at room temperature may make this technology well suited to support effective immunization campaigns for emerging pathogens on a global scale.