Current Research in Translational Medicine最新文献

{"title":"Allogeneic hematopoietic stem cell transplantation in 18 patients with atypical inherited bone marrow failure syndromes: Efficacy and long-term outcomes","authors":"Yimin Wang, Jing Wang, Qianqian Xiao, Xiaolin Yu, Xiaochen Song, Wenjun Li, Lei Deng, Yixi Hou, Fang Zhou","doi":"10.1016/j.retram.2025.103519","DOIUrl":"10.1016/j.retram.2025.103519","url":null,"abstract":"","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103519"},"PeriodicalIF":3.2,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144167913","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anthracycline and arsenic trioxide-based regimens for the treatment of acute promyelocytic leukemia: thiamine levels monitoring importance","authors":"Reda Garidi, Hassina Aftisse, Djedjiga Si Tayeb, Chahrazad Benchchouk, Nadia Sari Hassoun, Assia Alem, Tamim Alsuliman","doi":"10.1016/j.retram.2025.103520","DOIUrl":"10.1016/j.retram.2025.103520","url":null,"abstract":"","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103520"},"PeriodicalIF":3.2,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144139341","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cytomegalovirus HLA antigen load as a new potential predictive factor after haploidentical stem cell transplantation","authors":"Antonio Milano ,&nbsp;Jacopo Mariotti ,&nbsp;Pietro Crivello ,&nbsp;Giulia Di Maggio ,&nbsp;Giorgia Cornacchini ,&nbsp;Giuliana Lando ,&nbsp;Silvano Rossini ,&nbsp;Marialuisa Lavitrano ,&nbsp;Stefania Bramanti ,&nbsp;Roberto Crocchiolo","doi":"10.1016/j.retram.2025.103518","DOIUrl":"10.1016/j.retram.2025.103518","url":null,"abstract":"<div><div>Among the complications occurring after allogeneic hematopoietic stem cell transplantation (HSCT), infection by human Cytomegalovirus (CMV) represents one of the most relevant in terms of morbidity/mortality and specific management is required by either monitoring viral load and administer anti-CMV therapies. Due to the physiological role of the Human Leucocyte Antigen (HLA) system in presenting foreign antigens to the adaptive immune system to enhance viral clearance, we measured here the HLA antigen load for two immunodominant CMV peptides and correlate with transplant outcome in 238 consecutive adult patients undergoing haploidentical HSCT at a single center.</div><div>Interestingly, a higher class I antigen load (i.e. above the median) for protein IE1 correlated with an inferior absolute incidence of CMV infection; moreover, a statistically significant correlation with lower non-relapse mortality and higher overall survival was observed (HR: 0.32, 0.12–0.84, <em>p</em> = 0.02; 0.34 (0.17–0.69, <em>p</em> = 0.003; respectively), with a protective effect in patients with high antigen load values.</div><div>Despite additional research is needed, our exploratory data support the role of the HLA polymorphism on CMV infection and survival after HSCT. The introduction of CMV antigen load as a new potential predictive factor might contribute to further define the post-transplant risk on an individualised basis.</div></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103518"},"PeriodicalIF":3.2,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144098323","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Multidimensional evaluation of CMV-specific T Cells: enhancing therapy through transcriptional insights","authors":"Changchang Sun ,&nbsp;Mingli Xu ,&nbsp;Min Yan ,&nbsp;Meiying Shen ,&nbsp;Xiaojian Han ,&nbsp;Hongbin Zhang ,&nbsp;Chao Hu ,&nbsp;Yingming Wang ,&nbsp;Wang Wang ,&nbsp;Aishun Jin ,&nbsp;Yingying Wang","doi":"10.1016/j.retram.2025.103517","DOIUrl":"10.1016/j.retram.2025.103517","url":null,"abstract":"<div><div>Adoptive immunotherapy with CMV-specific T cells (CMV-VSTs) has shown favorable efficacy and minimal adverse effects in clinical settings, serving as prophylaxis, preemptive and/or curative treatment for the restoration of CMV-specific immunity in patients after allogeneic hematopoietic stem cell transplantation. The establishment of a CMV-VST bank enables the prompt use of CMV-VSTs as off-the-shelf therapeutics. CMV-VSTs can be generated through cell culture or immunomagnetic selection based on IFN-γ secretion or multimer technology. In this study, we investigated the feasibility of generating CMV-VSTs via cell expansion after IFN-γ based immunomagnetic isolation, with the goal of establishing a good-quality, cost-effective local production approach. We also assessed the value of incorporating transcriptomic analysis into the current T cell evaluation framework. Our results demonstrate that good-quality CMV-VSTs can be produced using either autologous feeder cells or feeder cells from the rapid expansion protocol (REP), in combination with cytokines such as IL-2 or IL-7/IL-15. Phenotypic and functional analyses confirmed the consistent quality of the final T cell products and showed no significant differences across the various combinations of feeder cells and cytokines. However, transcriptomic analysis highlighted the advantages of using IL-7/IL-15 and autologous feeder cells. Collectively, our findings offer new insights into future strategies for the manufacturing of antigen-specific T cells and underscore the importance of comprehensive, multidimensional assessment in T cell-based immunotherapies.</div></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103517"},"PeriodicalIF":3.2,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143902345","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Organ transplantation in Africa: Confronting socioeconomic, cultural, and infrastructural hurdles","authors":"David B. Olawade ,&nbsp;Aderonke Odetayo ,&nbsp;Sheila Marinze ,&nbsp;Eghosasere Egbon ,&nbsp;Viviane Chinwah","doi":"10.1016/j.retram.2025.103516","DOIUrl":"10.1016/j.retram.2025.103516","url":null,"abstract":"<div><h3>Background</h3><div>Organ transplantation is a critical procedure offering life-saving treatment for patients with end-stage organ failure. In Africa, however, the accessibility and development of organ transplantation are severely hampered by numerous barriers. Socioeconomic disparities, inadequate healthcare infrastructure, legal and ethical gaps, cultural resistance, and the dual burden of infectious and non-communicable diseases are among the significant challenges faced. This review aims to comprehensively explore these barriers and propose actionable strategies to address them.</div></div><div><h3>Method</h3><div>A narrative review was conducted by searching electronic databases, including PubMed, Google Scholar, Scopus, and JSTOR. The review prioritized studies addressing the challenges of organ transplantation in Africa, focusing on socioeconomic factors, healthcare infrastructure, cultural beliefs, legal frameworks, and the impact of infectious and non-communicable diseases. Studies offering solutions tailored to the African context were also included.</div></div><div><h3>Results</h3><div>The review identified several key obstacles, including high costs of transplantation, a limited number of transplant centers, and a critical shortage of skilled healthcare professionals. Cultural beliefs and widespread misconceptions impede organ donation acceptance. Additionally, infectious and non-communicable diseases complicate the transplantation process and outcomes. Weak legal frameworks exacerbate the risks of organ trafficking and unethical practices, while low public awareness further undermines efforts to enhance organ donation rates.</div></div><div><h3>Conclusion</h3><div>Addressing these multifaceted challenges necessitates a comprehensive approach. Strengthening healthcare infrastructure, enhancing capacity-building programs, developing robust legal and ethical frameworks, and implementing targeted public education campaigns are critical for improving organ transplantation in Africa.</div></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103516"},"PeriodicalIF":3.2,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143898484","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A novel complex mutation in the exon 9 of CALR gene: p.E381fs*52 (gagg_CCTCTTTGCCTC)","authors":"Alessandro Laganà,&nbsp;Claudia Ielo,&nbsp;Sonia Buffolino,&nbsp;Attilio Di Lascio,&nbsp;Luisa Bizzoni,&nbsp;Emilia Scalzulli,&nbsp;Ida Carmosino,&nbsp;Maria Laura Bisegna,&nbsp;Daniela Diverio,&nbsp;Massimo Breccia","doi":"10.1016/j.retram.2025.103514","DOIUrl":"10.1016/j.retram.2025.103514","url":null,"abstract":"","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103514"},"PeriodicalIF":3.2,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143838285","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Therapeutic promise of CRISPR-Cas9 gene editing in sickle cell disease and β-thalassemia: A current review","authors":"Hassan H. Almasoudi","doi":"10.1016/j.retram.2025.103513","DOIUrl":"10.1016/j.retram.2025.103513","url":null,"abstract":"<div><div>Sickle cell disease (SCD and β-thalassemia (BT) affects millions of people worldwide. In addition, around 500,000 infants are born with SCD and 60,000 people are diagnosed with BT every year. Mutations in the <em>hemoglobin subunit beta (HBB)</em> gene are responsible for causing both BT and SCD. Indeed, the diversity of potential mutations in the <em>HBB</em> gene elucidates the diversity in clinical severity observed in individuals with BT and related morbidities. On the other hand, SCD takes place because of the alteration in a single amino acid at position 6 in the beta-globin chain, where a base substitution occurs from glutamic acid to valine, which eventually results in abnormal sickle hemoglobin. Conventional therapies for BT and SCD including pharmaceutical drugs and blood transfusion might temporarily improve the clinical severity of these diseases, however these therapies cannot cure the diseases. CRISPR-Cas9 (CC9) is revolutionizing genome engineering, offering promising therapeutic avenues for genetic diseases. Therefore, CC9-mediated gene therapy provides great hope in the treatment of both BT and SCD. CC9-mediated gene therapy has already demonstrated its effectiveness in correcting both SCD and BT-causing mutations. Moreover, CC9-mediated gene editing was found to be effective in reactivating the expression of <em>hemoglobin F (HbF)</em> and regulating <em>LRF</em> and <em>BCL11A</em>. A number of clinical trials with CC9 gene-edited therapies are being carried out to elucidate their potential in treating BT and SCD. Genetics and pathophysiological mechanisms of SCD and BT, the mechanism of CC9-mediated gene editing, and common delivery methods of the CC9 system have been discussed in this review. Moreover, an in-depth discussion on applications and the current status of CC9-mediated gene editing in SCD and BT along with current challenges and future perspectives have been provided.</div></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103513"},"PeriodicalIF":3.2,"publicationDate":"2025-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143842882","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Perspectives on the use and availability of chimeric antigen receptor T cells (CAR-T) and cell therapies: A worldwide cross-sectional survey by the worldwide network for blood and marrow transplantation (WBMT)","authors":"Eddie HP Tan ,&nbsp;Mahmoud Aljurf ,&nbsp;Fazal Hussain ,&nbsp;Christian Chabannon ,&nbsp;Nina Worel ,&nbsp;Daniel Weisdorf ,&nbsp;Ibrahim Yakoub-Agha ,&nbsp;Sebastian Galeano ,&nbsp;Fermin Sanchez-Guijo ,&nbsp;Laurent Garderet ,&nbsp;Yoshiko Atsuta ,&nbsp;Annalisa Ruggeri ,&nbsp;Nada Hamad ,&nbsp;Sharukh Hashmi ,&nbsp;Cristobal Frutos ,&nbsp;Yoshihisa Kodera ,&nbsp;Adriana Seber ,&nbsp;Carmem Bonfim ,&nbsp;Dietger Niederwieser ,&nbsp;Damiano Rondelli ,&nbsp;Mickey BC Koh","doi":"10.1016/j.retram.2025.103515","DOIUrl":"10.1016/j.retram.2025.103515","url":null,"abstract":"<div><div>Chimeric antigen receptor T cell therapy (CAR-T) cells represent a new generation of autologous, allogeneic and personalised cell-based therapies that have revolutionised the treatment of B cell haematological malignancies. Despite their significant effectiveness in treating challenging relapsed and refractory diseases, access to this cutting-edge treatment remains a critical issue globally, even in high income countries. To gain insights into these challenges, the Worldwide Network for Blood &amp; Marrow Transplantation (WBMT) initiated a survey focused on the state of CAR-T and cellular therapy availability worldwide. The survey aimed to identify the accessibility, manufacturing capabilities, apheresis, accreditation, reimbursement, presence of regulatory frameworks and legal oversight of these cell-based therapies.</div><div>The survey included questions on demographics, the respondent's centre, CAR-T availability, details about haematopoietic stem cell transplant programs, supply and indications for CAR-T, quality assurance, and information about other cell and gene therapy products beside CAR-T. Conducted online over three months in 2023, the survey garnered 181 complete responses from various geographical regions, from North America, Asia, Europe, South and Central America, Australia and New Zealand, and Africa.</div><div>Our findings suggested a promising level of awareness and interest in CAR-T therapy globally, even in lower-income regions. However, survey respondents cited cost as the primary barrier to access, alongside infrastructure and governmental support issues. The survey also highlighted the varying reimbursement strategies across regions, with costs in Europe and North America being relatively similar while Asia showed more variability. There was also variability in the regulatory and accreditation frameworks associated with delivery of these novel therapies</div><div>As CAR-T therapy continues to grow, innovative solutions such as global partnerships, in-house production, and the establishment of cellular therapy centres in developing countries are essential. Addressing the challenges of access requires a comprehensive approach that combines efforts to lower costs, enhance healthcare infrastructure, and foster international collaborations, ensuring that CAR-T therapy becomes available to all who need it.</div></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 2","pages":"Article 103515"},"PeriodicalIF":3.2,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143850346","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acute-onset chronic inflammatory demyelinating polyneuropathy in a 23-year-old male exacerbated by an asymptomatic COVID-19 infection","authors":"Sawsan Ismail ,&nbsp;Aiman Abo Al shamat ,&nbsp;Ali Ghalion ,&nbsp;Hanafi Alyman Hannouf ,&nbsp;Tamim Alsuliman ,&nbsp;Kanaan Al-Tameemi","doi":"10.1016/j.retram.2025.103511","DOIUrl":"10.1016/j.retram.2025.103511","url":null,"abstract":"","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103511"},"PeriodicalIF":3.2,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143785894","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immune cell dysfunction: A critical player in development of diabetes complications","authors":"Mohamed J. Saadh ,&nbsp;Omer Qutaiba B. Allela ,&nbsp;Radhwan Abdul Kareem ,&nbsp;Ashishkumar Kyada ,&nbsp;H. Malathi ,&nbsp;Deepak Nathiya ,&nbsp;Deepak Bhanot ,&nbsp;Hayder Naji Sameer ,&nbsp;Atheer Khdyair Hamad ,&nbsp;Zainab H. Athab ,&nbsp;Mohaned Adil","doi":"10.1016/j.retram.2025.103510","DOIUrl":"10.1016/j.retram.2025.103510","url":null,"abstract":"<div><div>Diabetes mellitus, a global health challenge, influences millions worldwide by leading to severe complications and premature death. A key factor in its pathogenesis is immune cell dysfunction, which aggravates both type 1 and type 2 diabetes. The important role that immune cell dysregulation plays in the emergence of diabetes complications is investigated in this research. It highlights the manner in which diabetes compromises the immune system's adaptive as well as innate responses. Key defects in innate immunity include impaired pathogen recognition, and dysfunctional behavior of macrophages, neutrophils, and natural killer (NK) cells. Additionally, the complement system is dysregulated, and cytokine production is altered, affecting overall immune signaling. The study investigates the dysfunction of several T and B cell subsets, such as CD4+ T cells, CD8+ T cells, regulatory T cells, and B cells, in relation to adaptive immunity. These dysfunctions collectively contribute to chronic inflammation, reduced pathogen clearance, and increased susceptibility to infections, ultimately exacerbating diabetes complications. Developing targeted therapies to reduce diabetes complications and enhance patient outcomes requires an understanding of these mechanisms.</div></div>","PeriodicalId":54260,"journal":{"name":"Current Research in Translational Medicine","volume":"73 3","pages":"Article 103510"},"PeriodicalIF":3.2,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143918500","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}