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23 pages, 487 KB  
Article
Role of the Pharmacist in Supporting the Use of Connected Health Devices: Example of Connected Watches
by Cordélia Salomez-Ihl, Léa Liaigre, Wiceme Dala, Ambre Davat, Maud Barbado, Sébastien Chanoine, Philippe Py, Delphine Schmitt, Pascal Defaye and Pierrick Bedouch
Pharmacy 2026, 14(1), 28; https://doi.org/10.3390/pharmacy14010028 - 3 Feb 2026
Abstract
The use of Connected Medical Devices (CMDs) is growing significantly throughout the world. Although they are not dispensed in pharmacies and are not part of the pharmacy-only drug dispensing system, clinical pharmacists must be able to support patients in the use of these [...] Read more.
The use of Connected Medical Devices (CMDs) is growing significantly throughout the world. Although they are not dispensed in pharmacies and are not part of the pharmacy-only drug dispensing system, clinical pharmacists must be able to support patients in the use of these new technologies, which are central to their care. The aim of this study is to identify the role of the community pharmacist in supporting patients who use CMDs, using the case of connected watches in electrophysiology. Semi-structured interviews were conducted between 15 February and 20 April 2024 by a pharmacy student. The questionnaires were drafted in collaboration with a pharmacist, a cardiac electrophysiologist, a methodologist specializing in the evaluation of medical devices, and an ethical philosopher specializing in the support and acceptability of new technologies. The aim of these questionnaires was to study the use of connected watches and support for patients who own them. A total of 4 cardiac electrophysiologists and 10 cardiac electrophysiology patients were interviewed, and then 6 pharmacists were also questioned about the roles identified by physicians and patients. This study identified a major need on the part of specialist physicians for clinical pharmacist support in helping patients use connected watches. Patients expressed a high level of confidence in their pharmacists to support them, and in the motivation of pharmacists’ ability to take up these challenges. A number of challenges remain, such as the effective integration of this support into pharmacy practice, remuneration, and the organization of collaboration between clinical pharmacists and hospital electrophysiologists. Full article
(This article belongs to the Section Pharmacy Practice and Practice-Based Research)
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13 pages, 276 KB  
Article
School Leadership and the Association to Teachers’ Digital Competence in Supporting Students with Special Educational Needs
by Joacim Ramberg and Helena Hemmingsson
Educ. Sci. 2026, 16(2), 226; https://doi.org/10.3390/educsci16020226 - 2 Feb 2026
Abstract
The digitalisation of education has introduced new possibilities for inclusive teaching practices, particularly in supporting students with special educational needs (SEN). While digital tools have demonstrated potential to enhance learning outcomes and engagement for these students, the role of school leadership in fostering [...] Read more.
The digitalisation of education has introduced new possibilities for inclusive teaching practices, particularly in supporting students with special educational needs (SEN). While digital tools have demonstrated potential to enhance learning outcomes and engagement for these students, the role of school leadership in fostering teachers’ digital competence remains underexplored. The aim of the study is to investigate the association between school leadership, as rated by teachers, and teachers’ self-reported digital competence in supporting students with SEN. To this end, cross-sectional data from 285 Swedish teachers enrolled in special education training programmes have been used. The data were collected through the SELFIE survey, a European Commission tool designed to assess schools’ digital capacity. A stepwise linear regression analysis was conducted to examine the association between perceived school leadership and teachers’ self-reported digital competence in supporting students with SEN, controlling for teacher collaboration, infrastructure and equipment, and demographic variables. The results show a consistent and significant positive relationship between school leadership and teachers’ digital competence, even when other factors are accounted for. Teacher collaboration also contributed positively, though to a lesser extent, while infrastructure and equipment and demographic variables showed no significant effect. The study contributes knowledge by showing that teachers’ digital competence development depends not only on individual efforts but also on organisational factors, such as supportive school leadership, highlighting the importance of recognising school leadership as vital alongside digital resources in schools. Given the cross-sectional design, the findings should be interpreted cautiously and not as evidence of causal relationships. These findings suggest that school leadership is important in enabling teachers to use digital technologies to support students with SEN, highlighting practical and policy implications for strengthening school leadership in developing teachers’ digital competence in supporting students with SEN. Full article
(This article belongs to the Section Special and Inclusive Education)
21 pages, 1509 KB  
Article
Cross-Sectional Associations of Sport Participation, Academic Performance, and Psychological Well-Being Among Rural Romanian Adolescent Boys in the Context of Family Background
by Filoména Dávid, Krisztina Rácz and Pál Salamon
Children 2026, 13(1), 135; https://doi.org/10.3390/children13010135 - 16 Jan 2026
Viewed by 341
Abstract
Background: Adolescence is a sensitive period for psychological, academic, and social development, and sports participation has been described as a potential protective factor for academic performance and psychological well-being. However, limited research has examined the combined influence of sports involvement, sport type, and [...] Read more.
Background: Adolescence is a sensitive period for psychological, academic, and social development, and sports participation has been described as a potential protective factor for academic performance and psychological well-being. However, limited research has examined the combined influence of sports involvement, sport type, and family background on adolescents’ academic and psychological outcomes. This study aimed to investigate the associations between organized sport participation, sport type (football vs. judo), psychological well-being, psychosomatic symptoms, academic performance, and family socioeconomic background among adolescent boys. Methods: The sample consisted of 52 boys aged 11–14 years from a rural school, divided into football players (n = 13), judo athletes (n = 13), non-athletes (n = 13), and a contextual subgroup of students with special educational needs (SEN; n = 13), with the latter included for exploratory purposes only. Data included school-record-based academic performance and validated self-report measures of life satisfaction, depressive symptoms, psychosomatic complaints, perceived physical fitness, and socioeconomic background. Results: Athletes demonstrated significantly higher academic achievement than non-athletes in overall grade point average (p < 0.001), mathematics (p < 0.001), Romanian (p < 0.001), English (p = 0.03), and Hungarian (p < 0.001). They also reported higher life satisfaction (p < 0.001) but simultaneously showed slightly elevated depressive symptom scores (p < 0.001), indicating a paradoxical pattern of concurrent psychosocial benefits and psychological strain. Parental education (p < 0.001), parental occupational status (p = 0.01), and fathers’ occupational position (p = 0.02) were significantly higher among athletes’ families. Perceived physical fitness was also rated higher by athletes (p < 0.001). No significant differences were found in body mass index, family structure, or most psychosomatic symptoms. Conclusions: Sport participation was associated with more favorable academic and psychological indicators, yet also with elevated depressive symptoms, highlighting the dual nature of organized sport during adolescence. Future research should apply longitudinal designs, include female participants, and incorporate objective indicators of training load. Full article
(This article belongs to the Special Issue Physical Fitness and Health in Adolescents)
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15 pages, 544 KB  
Article
Preparation for Inclusive and Technology-Enhanced Pedagogy: A Cluster Analysis of Secondary Special Education Teachers
by Evaggelos Foykas, Eleftheria Beazidou, Natassa Raikou and Nikolaos C. Zygouris
Computers 2026, 15(1), 42; https://doi.org/10.3390/computers15010042 - 9 Jan 2026
Viewed by 293
Abstract
This study examines the profiles of secondary special education teachers regarding their readiness for inclusive teaching, with technology-enhanced practices operationalized through participation in STEAM-related professional development. A total of 323 teachers from vocational high schools and integration classes participated. Four indicators of professional [...] Read more.
This study examines the profiles of secondary special education teachers regarding their readiness for inclusive teaching, with technology-enhanced practices operationalized through participation in STEAM-related professional development. A total of 323 teachers from vocational high schools and integration classes participated. Four indicators of professional preparation were assessed: years of teaching experience, formal STEAM training, exposure to students with special educational needs (SEN), and perceived success in inclusive teaching, operationalized as self-reported competence in adaptive instruction, classroom management, positive attitudes toward inclusion, and collaborative engagement. Cluster analysis revealed three distinct teacher profiles: less experienced teachers with moderate perceived success and limited exposure to students with SEN; well-prepared teachers with high levels across all indicators; and highly experienced teachers with lower STEAM training and perceived success. These findings underscore the need for targeted professional development that integrates inclusive and technology-enhanced pedagogy through STEAM and is tailored to teachers’ experience levels. By integrating inclusive readiness, STEAM-related preparation, and technology-enhanced pedagogy within a person-centered profiling approach, this study offers actionable teacher profiles to inform differentiated professional development in secondary special education. Full article
(This article belongs to the Special Issue STEAM Literacy and Computational Thinking in the Digital Era)
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15 pages, 723 KB  
Article
Understanding Teachers’ Intention and Behaviour Towards Inclusive Education in Ghana: Applying the Theory of Planned Behaviour
by Michael Amponteng, Danielle Tracey and William Nketsia
Educ. Sci. 2026, 16(1), 93; https://doi.org/10.3390/educsci16010093 - 8 Jan 2026
Viewed by 396
Abstract
United Nations Sustainable Development Goal 4 advocates for equitable access to and participation in quality inclusive education for all learners. Inclusive education has gained worldwide recognition for promoting equity and social justice for students with special educational needs. Although the existing literature acknowledges [...] Read more.
United Nations Sustainable Development Goal 4 advocates for equitable access to and participation in quality inclusive education for all learners. Inclusive education has gained worldwide recognition for promoting equity and social justice for students with special educational needs. Although the existing literature acknowledges the significant role of teachers’ intention and behaviour towards the successful implementation of inclusive education, this area is under-researched in Sub-Saharan countries, including Ghana. In this study, applying the theory of planned behaviour (TPB), 484 teachers at pilot inclusive schools completed an online survey assessing the factors predicting their intention and teaching behaviour towards inclusive education. A path analysis of the TPB variables revealed that only attitude and self-efficacy significantly predicted the teachers’ intention to teach in inclusive classrooms. Moreover, both self-efficacy and intention were found to significantly predict inclusive behaviour. This study’s findings will strengthen the national commitment to implementing inclusive education policy and guide future research aimed at improving and expanding inclusive education in Ghana. Full article
(This article belongs to the Special Issue Teachers and Teaching in Inclusive Education)
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26 pages, 401 KB  
Article
Students with Autism in Spain: Key Attitudes and Competences for Inclusion
by M. Mercedes Arias-Pastor, Alejandra Bolado-Peña, Steven Van Vaerenbergh and Jerónimo J. González-Bernal
Educ. Sci. 2026, 16(1), 64; https://doi.org/10.3390/educsci16010064 - 3 Jan 2026
Viewed by 509
Abstract
Inclusive education aims to ensure equitable access to quality education for all students, including those with Autism Spectrum Disorder (ASD). This research analyzes the attitudes and competences of non-university Spanish teachers regarding the inclusion of students with ASD, considering relevant variables in previous [...] Read more.
Inclusive education aims to ensure equitable access to quality education for all students, including those with Autism Spectrum Disorder (ASD). This research analyzes the attitudes and competences of non-university Spanish teachers regarding the inclusion of students with ASD, considering relevant variables in previous studies such as teacher training, prior experience, and educational context, among others. The study involved a sample of 2310 teachers in active service during the 2023–2024 school year. Data collection was conducted through two questionnaires: the INTEA-EDG questionnaire, designed to measure teachers’ beliefs and attitudes towards the inclusion of students with ASD, and the CEFI-R questionnaire, which evaluates teachers’ competencies and skills for inclusive education. The results reflect a generally positive attitude towards the inclusion of students with ASD in mainstream schools, although significant variations were found depending on variables such as previous experience, teaching specialization, and specific training received. Based on these and other findings, the study highlights the need to strengthen initial and continuous training in inclusive education and attention to diversity of students with ASD, promote early positive contact with students with ASD, and foster collaborative work and the design of inclusive learning environments. Full article
29 pages, 5334 KB  
Article
A Preliminary Usability Study of a Novel Educational Training System to Teach ScratchJr. in School
by María Jesús Manzanares, Diana Pérez Marín and Celeste Pizarro
Computers 2026, 15(1), 17; https://doi.org/10.3390/computers15010017 - 1 Jan 2026
Viewed by 260
Abstract
Teaching programming to children at an early age has been proven to be beneficial. Some research has focused on how to teach programming to children with special needs. According to Human–Computer Interaction, all users should be involved in the design of their systems [...] Read more.
Teaching programming to children at an early age has been proven to be beneficial. Some research has focused on how to teach programming to children with special needs. According to Human–Computer Interaction, all users should be involved in the design of their systems (including learning systems). However, evaluation procedures with young children are a complex task, which can be even harder when the young children have some special needs. This paper presents a preliminary usability study of a novel educational training system to teach ScratchJr. to young children. 48 neurotypical students between 6 and 7 years and 2 students with special needs in a pilot study were asked to use the system to find out whether they could complete an input/output activity and how it is related to their preferences. Full article
(This article belongs to the Special Issue Future Trends in Computer Programming Education)
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17 pages, 299 KB  
Article
Teachers’ Attitudes Toward Inclusion and the ICF: Evidence from a National Survey in Portugal
by Maria José Saragoça and Adelinda Araújo Candeias
Educ. Sci. 2026, 16(1), 44; https://doi.org/10.3390/educsci16010044 - 30 Dec 2025
Viewed by 352
Abstract
The “International Classification of Functioning, Disability and Health (ICF)” frames disability as an interaction between individual functioning and contextual factors, promoting participation over deficit-based models. In Portugal, the ICF was mandatory for eligibility decisions between 2008 and 2018 and remains a conceptual reference [...] Read more.
The “International Classification of Functioning, Disability and Health (ICF)” frames disability as an interaction between individual functioning and contextual factors, promoting participation over deficit-based models. In Portugal, the ICF was mandatory for eligibility decisions between 2008 and 2018 and remains a conceptual reference in inclusive education. However, little is known about teachers’ attitudes toward its use. This study explored Portuguese teachers’ attitudes toward (a) inclusion of students with “special educational needs (SEN)” and (b) the ICF as a framework for educational planning. A cross-sectional online survey was conducted with 417 public-school teachers working across all levels of compulsory education, from pre-school to secondary education. The questionnaire assessed six indicators of inclusion attitudes and a validated 27-item ICF Attitudes Scale. Data were analyzed using descriptive and correlational procedures. Teachers expressed strong support for participation in school life and selective academic integration, while rejecting full-time placement for all SEN students. Attitudes toward the ICF were generally positive, highlighting its perceived usefulness for planning and identifying barriers. Professional background variables showed only weak associations with these attitudes. Teachers value inclusive participation and recognize the ICF’s potential, but practical implementation depends on time, resources, and collaboration. Findings underscore the need for practice-based professional development to strengthen inclusive education. Full article
18 pages, 580 KB  
Article
Developing Sustainability Modules for Architectural Engineering: An Exploratory Study
by Faisal Alhassani and Muhammad Rakeh Saleem
Sustainability 2025, 17(23), 10772; https://doi.org/10.3390/su172310772 - 1 Dec 2025
Viewed by 638
Abstract
Despite growing emphasis on sustainability across engineering disciplines, empirical evidence on how structured interventions shape students’ sustainability knowledge and understanding remains limited. This study introduces and evaluates a set of purposefully designed sustainability modules integrated into an architectural engineering design studio. It addresses [...] Read more.
Despite growing emphasis on sustainability across engineering disciplines, empirical evidence on how structured interventions shape students’ sustainability knowledge and understanding remains limited. This study introduces and evaluates a set of purposefully designed sustainability modules integrated into an architectural engineering design studio. It addresses a persistent gap in student knowledge due to insufficient and non-coherent integration of sustainability topics. Noting the increased necessity to tackle complex sustainability challenges by systems thinking and applied design, the modules were designed to support learning across five progressive steps: foundational concepts, specialization and peer teaching, hands-on assessment, project-based integration, and reflective synthesis and future implementation. Using a mixed-methods approach, the modules were tested through pre- and post-intervention assessment, supported by statistical analysis (t = 41.92, p < 0.001) and qualitative feedback from students and instructors. The findings suggest significant improvements in students’ sustainability knowledge and in-depth engagement when incorporating active and collaborative learning strategies. Similarly, project-based learning and peer learning exercises were seen as most effective in fostering comprehension and applied understanding. The iterative approach of refining module’s content and delivery based on students’ feedback—such as incorporating reflective prompts in later sessions—improved conceptual clarity and strengthened student learning needs and relevance to the study topic. By addressing the gaps in knowledge and curriculum integration, this exploratory study offers a transformative framework to integrate sustainability into engineering curricula and highlights the importance of pedagogical strategies in promoting sustainability competencies within engineering. Full article
(This article belongs to the Special Issue Innovative Research Methods for Sustainable Educational Development)
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14 pages, 320 KB  
Article
Rethinking School Inclusion: A Comparative Analysis of Decree-Laws No. 3/2008 and No. 54/2018 in Portugal Based on UNESCO Reports
by Elsa Gabriel Morgado, Antonino Pereira, Luís Borges Gouveia, João Bartolomeu Rodrigues and Levi Leonido
Educ. Sci. 2025, 15(12), 1612; https://doi.org/10.3390/educsci15121612 - 29 Nov 2025
Viewed by 1026
Abstract
This article proposes a comparative analysis of Decree-Laws No. 3/2008 and No. 54/2018, legal instruments that define the models for supporting inclusive education in Portugal. By linking national regulatory developments with data and recommendations from UNESCO’s monitoring reports (GEM Reports), this research assesses [...] Read more.
This article proposes a comparative analysis of Decree-Laws No. 3/2008 and No. 54/2018, legal instruments that define the models for supporting inclusive education in Portugal. By linking national regulatory developments with data and recommendations from UNESCO’s monitoring reports (GEM Reports), this research assesses the structural and conceptual changes underlying Portuguese education policy. The research follows a qualitative approach, of an exploratory and interpretative nature, using comparative document analysis as the central method. The results reveal a transition from a model focused on categorizing students with special educational needs (SEN) to an approach focused on universally meeting the needs of all students. The Discussion and Conclusions and Recommendations highlight the need to strengthen teacher training, pedagogical leadership, and monitoring based on disaggregated data, as recommended by UNESCO. Full article
35 pages, 1954 KB  
Systematic Review
Exploring Artificial Intelligence in Inclusive Education: A Systematic Review of Empirical Studies
by Jiahui Li, Yuyang Yan and Xiaojun Zeng
Appl. Sci. 2025, 15(23), 12624; https://doi.org/10.3390/app152312624 - 28 Nov 2025
Cited by 1 | Viewed by 2014
Abstract
This systematic review synthesizes empirical evidence on the role of Artificial Intelligence (AI) in inclusive education. The review aimed to examine (1) the impact of AI technologies on learning outcomes and engagement among students with special needs, (2) barriers and enablers influencing AI [...] Read more.
This systematic review synthesizes empirical evidence on the role of Artificial Intelligence (AI) in inclusive education. The review aimed to examine (1) the impact of AI technologies on learning outcomes and engagement among students with special needs, (2) barriers and enablers influencing AI adoption by educators, and (3) the role of theoretical frameworks in guiding AI-based interventions. A comprehensive search was conducted in Scopus, Web of Science, DOAJ, and Google Scholar for English-language, peer-reviewed studies published between 2020 and 2025. Sixteen (16) studies met the inclusion criteria and were appraised using the Mixed Methods Appraisal Tool (MMAT). Findings indicate that AI tools enhance personalization, accessibility, and engagement, particularly for learners with disabilities, while barriers such as infrastructure gaps and low digital literacy persist. Enablers include institutional support and teacher training, though theoretical frameworks were inconsistently applied. Limitations include the exclusion of grey literature and reliance on short-term studies. AI can advance inclusive education when integrated with ethical, pedagogical, and institutional strategies, while future research should prioritize longitudinal, theory-driven, and culturally responsive models. Full article
(This article belongs to the Special Issue ICT in Education, 3rd Edition)
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25 pages, 993 KB  
Article
Parent Perceptions of Special Education and Gifted Education Services for Twice-Exceptional Students in Ireland
by Orla Dunne, Keri M. Guilbault, Leeanne Hinch, Aiswarya Radhakrishnan and Colm O’Reilly
Educ. Sci. 2025, 15(12), 1600; https://doi.org/10.3390/educsci15121600 - 27 Nov 2025
Viewed by 1619
Abstract
Twice-exceptional students, those who are both gifted and have an additional educational need, represent a complex and underserved population within education systems. While recognition of twice exceptionality has greatly increased in gifted education literature, little is known about the experiences of students and [...] Read more.
Twice-exceptional students, those who are both gifted and have an additional educational need, represent a complex and underserved population within education systems. While recognition of twice exceptionality has greatly increased in gifted education literature, little is known about the experiences of students and their families in Ireland, where no national policy or framework currently addresses their dual needs. This exploratory mixed-methods study aimed to examine the perspectives of 232 parents of twice-exceptional children who attended an enrichment summer programme for gifted students. Through an anonymous survey, the researchers investigated the frequency of specialised services provided for both giftedness and disabilities, as well as how satisfied parents were with these services. The findings indicated that, while two-thirds of students did receive special education support, the majority received no services focused on their giftedness. Parents expressed significantly higher dissatisfaction with gifted provisions than with special education, mentioning the lack of differentiation and access to advanced materials in class, as well as an emphasis on their child’s challenges, as opposed to their strengths. The study’s findings highlight substantial policy and practice gaps in Ireland’s current provision for twice-exceptional students and underscore the need for integrated support systems, teacher education, and inclusion of parent perspectives in educational planning. Full article
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24 pages, 1442 KB  
Article
Enhancing Student Motivation and Competencies via the WWH Teaching Method: A Case Study on the NoSQL Database Course
by Bin Yu, Yihong Liu, Yuhui Fan, Shaohua Liu, Xiaoyan Li and Ruoyu Li
Electronics 2025, 14(22), 4453; https://doi.org/10.3390/electronics14224453 - 14 Nov 2025
Viewed by 436
Abstract
NoSQL databases are vital for modern big data applications, yet traditional teaching methods struggle with lagging content, insufficient practice, and low student engagement. To address these issues, this paper proposes the WWH-integrated teaching method “Why learn, What learn, How learn” for a NoSQL [...] Read more.
NoSQL databases are vital for modern big data applications, yet traditional teaching methods struggle with lagging content, insufficient practice, and low student engagement. To address these issues, this paper proposes the WWH-integrated teaching method “Why learn, What learn, How learn” for a NoSQL database course. WWH combines three core approaches: the general–special method, which structures knowledge from foundational concepts to specialized technologies; the comparative method, which contextualizes NoSQL value via real-scenario analysis; and the theory–practice combination method, which links concepts to hands-on tasks, supplemented by the problem-guidance and key-highlighting strategies. A quasi-experiment with two cohorts (80 students each; 2023 cohort as control, 2024 as experimental) validated WWH. Quantitative results showed significant improvements: theoretical exam scores rose by 9.2 points (t(158) = 9.21, p < 0.001) and experimental scores by 10.3 points (t(158) = 7.92, p < 0.001), and classroom discussion rates increased from 45.2% to 82.7% (χ2(1) = 28.90, p < 0.001). Qualitative analysis of student essays and project reports further confirmed deeper conceptual understanding, stronger tradeoff awareness, and enhanced knowledge integration in the experimental cohort. This study provides an evidence-based, student-centered framework for modernizing NoSQL instruction, better preparing students for industry data management needs. Full article
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26 pages, 1233 KB  
Article
Service Learning Projects and CFS-IRA Principles: Application to the Food Bank Chair from the Working with People Model
by Priscila Nole Correa, Irely Joelia Farías Estrada, Guillermo Aliaga and Claudia Zuluaga
Sustainability 2025, 17(22), 10212; https://doi.org/10.3390/su172210212 - 14 Nov 2025
Cited by 1 | Viewed by 541
Abstract
This research study addresses the critical contradiction within global food systems: unsustainable consumption patterns and persistent food insecurity coexist and are exacerbated by food waste, which deepens socioeconomic inequalities and generates negative environmental externalities. In this scenario, higher education plays a central role [...] Read more.
This research study addresses the critical contradiction within global food systems: unsustainable consumption patterns and persistent food insecurity coexist and are exacerbated by food waste, which deepens socioeconomic inequalities and generates negative environmental externalities. In this scenario, higher education plays a central role in adopting comprehensive strategic frameworks to develop specialized human capital and influence society. This study analyzes a Service Learning model that integrates the CFS-IRA Principles to promote the SDGs and ensure responsible consumption. Based on a case study of the Food Bank Chair spanning 10 years and 212 projects, the implementation of this model was evaluated using the Working with People (WWP) method, which combines the development of postgraduate students’ skills with community service to address social problems. The results demonstrated the effectiveness of the SL-WWP model in strengthening students’ technical, social, and ethical competencies while reducing food waste. The evaluation showed strong alignment with key SDGs, with outstanding performance in governance, although the need to strengthen environmental and social criteria was identified. The originality lies in integrating the CFS-IRA Principles into an SL model that encourages innovative cooperation among universities, civil society, and public–private sectors, offering a replicable proposal for higher education institutions to establish themselves as agents of change towards sustainability. Full article
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28 pages, 873 KB  
Review
Curing Sickle Cell Disease by Allogeneic Hematopoietic Stem Cell (HSC) Transplantation Toward In Vivo HSC Gene Therapy
by Rina Kansal
Genes 2025, 16(11), 1367; https://doi.org/10.3390/genes16111367 - 11 Nov 2025
Viewed by 2194
Abstract
Sickle cell disease comprises a group of prevalent inherited disorders defined by an underlying sickle cell allele that forms sickle hemoglobin. The incidence of this disease is rising, with more than 500,000 children born with it globally. The disease carries significant morbidity and [...] Read more.
Sickle cell disease comprises a group of prevalent inherited disorders defined by an underlying sickle cell allele that forms sickle hemoglobin. The incidence of this disease is rising, with more than 500,000 children born with it globally. The disease carries significant morbidity and mortality. Its only curative treatment was an allogeneic hematopoietic stem cell (HSC) transplant (HSCT) until late 2023, when two one-time gene therapies were approved for treating patients aged 12 years or older with severe sickle cell disease. This work aims to inform readers about these two gene therapies: one lentiviral-based and the other nonviral. The latter is based on the Nobel Prize-winning discovery of clustered, regularly interspaced, short, palindromic repeats (CRISPR)/CRISPR-associated (Cas)9 proteins and single-guide RNA (sgRNA)-based genome editing. Both approved gene therapies require an autologous HSCT with ex vivo genetically edited autologous hematopoietic stem and progenitor cells. Therefore, access to these gene therapies is limited to specialized centers with expertise in HSCTs. This review is meant for students, researchers, and clinical practitioners. It explains the basis for both approved gene therapies, their mechanisms of action, differences, risks, and other lentiviral-based and CRISPR-Cas9-based ex vivo gene therapies for sickle cell disease in clinical development. Additionally, it discusses the current state of preclinical studies for in vivo HSC gene therapy for sickle cell disease, which utilize advanced genome editing technologies developed after CRISPR-Cas9-sgRNA-based genome editing. In vivo HSC gene therapy, after it is clinically developed, would eliminate the need for an HSCT in receiving gene therapy and vastly increase access for numerous patients worldwide, even in low-income countries with the most significant disease burden. Full article
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