Molecular TherapyPub Date : 2025-07-16DOI: 10.1016/j.ymthe.2025.07.013
Coral M Kasden,Maria P Corzo,Christopher D Greer,Tanvi Singh,Rufranshell Reyes,Alaine Castillo,Zoltan Arany,Benjamin W Kozyak,Hansell H Stedman
{"title":"AAV-microutrophin gene therapy confers long-term cardioprotection against pharmacologic and exercise-induced injury in dystrophin deficiency.","authors":"Coral M Kasden,Maria P Corzo,Christopher D Greer,Tanvi Singh,Rufranshell Reyes,Alaine Castillo,Zoltan Arany,Benjamin W Kozyak,Hansell H Stedman","doi":"10.1016/j.ymthe.2025.07.013","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.07.013","url":null,"abstract":"Duchenne muscular dystrophy (DMD) is the most common childhood-onset muscle degenerative disease, caused by genetic deficiency of dystrophin, resulting in premature death due to cardiorespiratory failure. Gene therapy clinical trials employing adeno-associated virus (AAV) systemically delivering miniaturized dystrophin have provided mixed results with lingering concerns about safety, long-term efficacy, and the absence of information about cardiac protection. AAV-microutrophin (AAV-μUtro) represents an attractive alternative, previously shown in pre-clinical models to rescue skeletal muscle disease without stimulating an immune response to transgene product. It remains untested whether AAV-μUtro is protective in the dystrophic heart, in part due to limitations in DMD cardiomyopathy models. Here we present reproducible pharmacologic- and exercise-induced cardiac injury models in the mdx mouse and rigorous testing of AAV-μUtro for efficacy and durability. AAV-μUtro was highly protective against cardiac injury in these distinct models, evidenced by reduced cardiac troponin I levels and Evans Blue Dye uptake compared to untreated mdx mice. AAV-μUtro improved running performance following a cardiac stressor and prevented maladaptive cardiac remodeling in response to daily running 10 months-post-treatment. These results demonstrate AAV-μUtro's durable cardioprotection during cardiac stress and daily physical activity, and are promising with respect to meaningful amelioration of cardiac disease in children with DMD.","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"14 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-15DOI: 10.1016/j.ymthe.2025.07.002
Yunyi Liu,Yatao Wu,Changyue Yuan,Bei Hu,Yuxi Xu,Hailong Ou,Juan Li,Dan Qi,Bi Shi,Yiliang Wu,Jason H Huang,Erxi Wu,Xiaoxiao Hu
{"title":"Disruption of the Nucleolin-CXCR4 Interaction by the DNA Aptamer HY-4 Halts Colorectal Cancer Metastasis.","authors":"Yunyi Liu,Yatao Wu,Changyue Yuan,Bei Hu,Yuxi Xu,Hailong Ou,Juan Li,Dan Qi,Bi Shi,Yiliang Wu,Jason H Huang,Erxi Wu,Xiaoxiao Hu","doi":"10.1016/j.ymthe.2025.07.002","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.07.002","url":null,"abstract":"Colorectal cancer, characterized by its aggressive metastatic behavior and often poor prognosis, urgently necessitates improved diagnostic and therapeutic strategies. This study introduces HY-4, an aptamer developed via a non-SELEX method, which shows exceptional specificity and affinity for nucleolin (NCL), a highly expressed oncogenic protein in various cancers. With remarkable biocompatibility, HY-4 significantly reduces the migration and invasion of LoVo cancer cells by disrupting the NCL-CXCR4 interaction, a pivotal pathway in cancer metastasis. This disruption highlights the potential of HY-4 to inhibit cancer metastasis without producing adverse effects. Notably, HY-4 distinguishes itself from conventional NCL-targeting aptamers by avoiding the typical G-quadruplex structure and instead adopting a unique non-G-quadruplex conformation to create a novel binding site. This unique structural conformation provides critical insight into NCL-targeting mechanisms and could profoundly influence the landscape of targeted cancer therapy. By utilizing advanced techniques such as circular dichroism spectroscopy and molecular docking simulations, we obtained detailed insights into the structural dynamics and inhibitory interactions of HY-4, thereby deepening our understanding of the crucial structure-activity relationships in NCL-targeted drug development. In summary, HY-4 represents a promising advancement in targeted therapy, potentially heralding a paradigm shift in the treatment and diagnosis of colorectal cancer.","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"11 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2025-07-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144645839","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-09DOI: 10.1016/j.ymthe.2025.06.043
Min Liu,Fan Yi
{"title":"IGFBP7: A potential target for tubular lipogenesis in CKD.","authors":"Min Liu,Fan Yi","doi":"10.1016/j.ymthe.2025.06.043","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.06.043","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"7 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144603362","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-05DOI: 10.1016/j.ymthe.2025.06.046
Joan Roig-Soriano, Ángel Edo, Sergi Verdés, Carlos Martín-Alonso, Cristina Sánchez-de-Diego, Laura Rodriguez-Estevez, Antonio L Serrano, Carmela R Abraham, Assumpció Bosch, Francesc Ventura, Bryen A Jordan, Pura Muñoz-Cánoves, Miguel Chillón
{"title":"Long-term effects of s-KL treatment in wild-type mice: Enhancing longevity, physical well-being, and neurological resilience.","authors":"Joan Roig-Soriano, Ángel Edo, Sergi Verdés, Carlos Martín-Alonso, Cristina Sánchez-de-Diego, Laura Rodriguez-Estevez, Antonio L Serrano, Carmela R Abraham, Assumpció Bosch, Francesc Ventura, Bryen A Jordan, Pura Muñoz-Cánoves, Miguel Chillón","doi":"10.1016/j.ymthe.2025.06.046","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.06.046","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":""},"PeriodicalIF":12.1,"publicationDate":"2025-07-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144575850","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-02Epub Date: 2025-03-31DOI: 10.1016/j.ymthe.2025.03.048
Tobias Tix, Mohammad Alhomoud, Roni Shouval, Gloria Iacoboni, Edward R Scheffer Cliff, Doris K Hansen, Saad Z Usmani, Gilles Salles, Miguel-Angel Perales, David M Cordas Dos Santos, Kai Rejeski
{"title":"Non-relapse mortality with bispecific antibodies: A systematic review and meta-analysis in lymphoma and multiple myeloma.","authors":"Tobias Tix, Mohammad Alhomoud, Roni Shouval, Gloria Iacoboni, Edward R Scheffer Cliff, Doris K Hansen, Saad Z Usmani, Gilles Salles, Miguel-Angel Perales, David M Cordas Dos Santos, Kai Rejeski","doi":"10.1016/j.ymthe.2025.03.048","DOIUrl":"10.1016/j.ymthe.2025.03.048","url":null,"abstract":"<p><p>Bispecific antibodies (BsAb) are associated with distinct immune-related toxicities that impact morbidity and mortality. This systematic review and meta-analysis examined non-relapse mortality (NRM) with BsAb therapy in B-cell non-Hodgkin lymphoma (NHL) and multiple myeloma (MM). A PubMed and Embase search up to October 2024 identified 29 studies (21 NHL, 8 MM) involving 2,535 patients. The overall NRM point estimate was 4.7% (95% confidence interval [CI] 3.4%-6.4%), with a median follow-up of 12.0 months. We noted no significant difference in NRM across disease entities (NHL: 4.2%, MM: 6.2%, p = 0.22). In NHL, prespecified subgroup analyses revealed increased NRM in real-world studies compared to clinical trials. For MM, an association between NRM and higher response rates and longer follow-up was noted. Meta-regression comparing BsAb and CAR-T therapies (n = 8,592) showed no significant NRM difference when accounting for key study-level confounders (p = 0.96). Overall, infections were the leading cause of NRM, accounting for 71.8% of non-relapse deaths. Of the infection-related deaths, 48% were attributed to COVID-19. In a pre-specified sensitivity analysis excluding COVID-19 fatalities, the overall NRM estimate was 3.5% (95% CI 2.6%-4.6%). Taken together, these results provide a benchmark for the estimated NRM with BsAb therapy and highlight the paramount importance of infection reporting, prevention, and mitigation.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"3163-3176"},"PeriodicalIF":12.1,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12266037/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143764456","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-02Epub Date: 2025-03-13DOI: 10.1016/j.ymthe.2025.03.019
Alexandra Dreyzin, Lipei Shao, Yihua Cai, Kyu Lee Han, Michaela Prochazkova, Michael Gertz, Bonnie Yates, Rongye Shi, Kathryn Martin, Naomi Taylor, Steven Highfill, Maura O'Neill, Thorkell Andresson, David Stroncek, Ping Jin, Nirali N Shah
{"title":"Immunophenotype of CAR T cells and apheresis products predicts response in CD22 CAR T cell trial for B cell acute lymphoblastic leukemia.","authors":"Alexandra Dreyzin, Lipei Shao, Yihua Cai, Kyu Lee Han, Michaela Prochazkova, Michael Gertz, Bonnie Yates, Rongye Shi, Kathryn Martin, Naomi Taylor, Steven Highfill, Maura O'Neill, Thorkell Andresson, David Stroncek, Ping Jin, Nirali N Shah","doi":"10.1016/j.ymthe.2025.03.019","DOIUrl":"10.1016/j.ymthe.2025.03.019","url":null,"abstract":"<p><p>Although CAR T cell therapy is increasingly used to treat relapsed B cell acute lymphoblastic leukemia (ALL), 20%-30% of patients do not respond, and few clinical predictors of response have been established, especially in the pediatric population. A deeper analysis of CAR T cell infusion products, along with the apheresis product used as the starting material for CAR T cell manufacturing, provides valuable insights for predicting clinical outcomes. We analyzed infusion products and CD4/8-selected T cell starting materials from pediatric and young adult patients on a single-center study with relapsed/refractory B cell ALL who were undergoing treatment with CD22 CAR T cells and evaluated differences between T cells from responders and non-responders (NCT023215612). We found that CAR T cells from non-responders had a more differentiated T cell phenotype and overexpressed genes associated with cytotoxicity and exhaustion compared with those of responders. Furthermore, we found that these differences could be tracked back to the apheresis materials prior to CAR T cell manufacturing. Using flow cytometry-based immunophenotypic markers, we developed a scoring system that distinguished non-responders based on T cell phenotype at the time of apheresis. These findings can help inform outcomes for patients and providers as well as provide insights into targeted manufacturing changes to optimize CAR T cell efficacy.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"3360-3374"},"PeriodicalIF":12.1,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12265959/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143634244","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-02Epub Date: 2025-06-25DOI: 10.1016/j.ymthe.2025.06.003
Giovanna Giacca, Mario Leonardo Squadrito
{"title":"Ad-justing macrophages for cancer immunotherapy.","authors":"Giovanna Giacca, Mario Leonardo Squadrito","doi":"10.1016/j.ymthe.2025.06.003","DOIUrl":"10.1016/j.ymthe.2025.06.003","url":null,"abstract":"","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"2972-2974"},"PeriodicalIF":12.1,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12266015/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144506875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-02Epub Date: 2025-03-08DOI: 10.1016/j.ymthe.2025.03.003
Bhavya S Doshi, Caroline A Markmann, Noelle Novak, Silvia Juarez Rojas, Robert Davidson, Julia Q Chau, Wei Wang, Sean Carrig, Cristina Martos Rus, Benjamin J Samelson-Jones, Juliana C Small, Vijay G Bhoj, Lindsey A George
{"title":"Use of CD19-targeted immune modulation to eradicate AAV-neutralizing antibodies.","authors":"Bhavya S Doshi, Caroline A Markmann, Noelle Novak, Silvia Juarez Rojas, Robert Davidson, Julia Q Chau, Wei Wang, Sean Carrig, Cristina Martos Rus, Benjamin J Samelson-Jones, Juliana C Small, Vijay G Bhoj, Lindsey A George","doi":"10.1016/j.ymthe.2025.03.003","DOIUrl":"10.1016/j.ymthe.2025.03.003","url":null,"abstract":"<p><p>Neutralizing antibodies (NAbs) against adeno-associated virus (AAV) represent a significant obstacle to the efficacy of systemic recombinant AAV vector administration or re-administration. While there are some promising preclinical immunomodulation strategies in development, insights into which B cell subsets and compartments maintain persistent AAV NAb may define the optimal eradication strategy. Given the limited success of CD20-directed monotherapy in previous studies, we hypothesized that CD19-directed approaches that extend targeting into the plasma cell compartments may improve AAV NAb eradication. We tested this approach in mice using chimeric antigen receptor T (CAR-T) cells or monoclonal antibodies (mAbs). We observed that combination mAbs targeting CD19, CD22, CD20, or B220 in mice did not eliminate tissue-resident B cells and, correspondingly, did not deplete pre-existing high titer AAV8 NAb. In contrast, CD19 CAR-T therapy eliminated peripheral and tissue-resident B cells and plasma cells and resulted in a marked reduction or eradication of high titer AAV8 NAb that permitted successful transgene expression following systemic AAV8 re-administration in mice. This successful therapeutic approach in mice identifies the population and location of B cells necessary to reduce or eradicate AAV NAb sufficiently to permit successful transgene expression with systemic AAV vector administration.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"3073-3085"},"PeriodicalIF":12.1,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12265964/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-02Epub Date: 2025-03-25DOI: 10.1016/j.ymthe.2025.03.027
Paul R Sargunas, Emily Ariail, Raquel Lima E Silva, Akash Patil, Mingliang Zhang, Jikui Shen, Beatriz Silva Lopes, Yuseong Oh, Amelia C McCue, Ranjani Ramasubramanian, A Carson Stephenson, Aleksander S Popel, Peter A Campochiaro, Jamie B Spangler
{"title":"Bispecific receptor decoy proteins block ocular neovascularization via simultaneous blockade of vascular endothelial growth factor A and C.","authors":"Paul R Sargunas, Emily Ariail, Raquel Lima E Silva, Akash Patil, Mingliang Zhang, Jikui Shen, Beatriz Silva Lopes, Yuseong Oh, Amelia C McCue, Ranjani Ramasubramanian, A Carson Stephenson, Aleksander S Popel, Peter A Campochiaro, Jamie B Spangler","doi":"10.1016/j.ymthe.2025.03.027","DOIUrl":"10.1016/j.ymthe.2025.03.027","url":null,"abstract":"<p><p>Several debilitating eye diseases that lead to vision loss are driven by ocular neovascularization, which entails abnormal blood vessel growth in the eye. Neovascularization is often induced by the upregulation of vascular endothelial growth factor (VEGF) ligands, which activate angiogenesis through engagement of VEGF receptor (VEGFR) proteins on endothelial cells. Therapeutic interventions that block ocular neovascularization by targeting VEGF ligands, particularly VEGF-A, have revolutionized eye disease treatment. However, a significant population of patients are either non-responders or develop resistance, which can be driven by the upregulation of other VEGF family ligands such as VEGF-C. Here, we engineered two bispecific receptor decoy fusion proteins that incorporate domains of VEGFR-1 and VEGFR-2 for more effective and comprehensive inhibition of VEGF ligands. We demonstrated that our engineered proteins bind all VEGF ligands and can sequester two ligands simultaneously. We further showed that these molecules block VEGF activity to potently inhibit proliferation, migration, and survival of human endothelial cells. Moreover, these receptor decoy proteins significantly reduced ocular neovascularization in two mouse models at doses wherein the current standard-of-care anti-VEGF therapy is ineffective. Collectively, our engineered receptor decoy proteins present a new architecture for VEGF pathway inhibition, offering a promising treatment paradigm for ocular diseases.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"3128-3146"},"PeriodicalIF":12.1,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12265965/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143720362","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Molecular TherapyPub Date : 2025-07-02Epub Date: 2025-04-05DOI: 10.1016/j.ymthe.2025.04.005
Zhaohui Liang, Suresh Kanna Murugappan, Yuxuan Li, Man Nga Lai, Yajing Qi, Yi Wang, Ho Yin Edwin Chan, Marianne M Lee, Michael K Chan
{"title":"Gene delivery of SUMO1-derived peptide rescues neuronal degeneration and motor deficits in a mouse model of Parkinson's disease.","authors":"Zhaohui Liang, Suresh Kanna Murugappan, Yuxuan Li, Man Nga Lai, Yajing Qi, Yi Wang, Ho Yin Edwin Chan, Marianne M Lee, Michael K Chan","doi":"10.1016/j.ymthe.2025.04.005","DOIUrl":"10.1016/j.ymthe.2025.04.005","url":null,"abstract":"<p><p>Developing α-synuclein aggregation inhibitors is challenging because its aggregation process involves several microscopic steps and heterogeneous intermediates. Previously, we identified a SUMO1-derived peptide, SUMO1(15-55), that exhibits tight binding to monomeric α-synuclein via SUMO-SUMO-interacting motif (SIM) interactions, and effectively blocks the initiation of aggregation and formation of toxic aggregates in vitro. In cellular and Drosophila models, SUMO1(15-55) was efficacious in protecting neuronal cells from α-synuclein-induced neurotoxicity and neuronal degeneration. Given the demonstrated ability of SUMO1(15-55) to sequester α-synuclein monomers thereby blocking oligomer formation, we sought to evaluate whether it could be equally effective against the aggregation-prone familial mutant α-synuclein-A53T. Herein, we show that SUMO1(15-55) selectively binds to monomeric α-synuclein-A53T, inhibits primary nucleation, and prevents the formation of structured protofibrils in vitro, thereby protecting neuronal cells from protofibril-induced cell death. We further demonstrate that larval feeding of a designed His<sub>10</sub>-SUMO1(15-55) that exhibits enhanced sub-stoichiometric suppression of α-synuclein-A53T aggregation in vitro can ameliorate Parkinson's disease (PD)-related symptoms in α-synuclein-A53T transgenic Drosophila models, while its rAAV-mediated gene delivery can relieve the PD-related histological and behavioral deficiencies in an rAAV-α-synuclein-A53T mouse PD model. Our findings suggest that gene delivery of His<sub>10</sub>-SUMO1(15-55) may serve as a clinical therapy for a spectrum of α-synuclein-aggregation associated synucleinopathies.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"3056-3072"},"PeriodicalIF":12.1,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12266022/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143795762","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}