Molecular Therapy最新文献_第6页

Harnessing engineered NK cells for refractory CD30+ lymphoma. 利用工程NK细胞治疗难治性CD30+淋巴瘤。

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-24 DOI: 10.1016/j.ymthe.2025.06.001

Yago Nieto,Katayoun Rezvani

引用次数: 0

Double trouble: Non-relapse mortality with bispecific antibodies in lymphoma and myeloma. 双重困扰：淋巴瘤和骨髓瘤双特异性抗体的非复发死亡率。

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-24 DOI: 10.1016/j.ymthe.2025.06.014

Rahul Banerjee,Samuel Yamshon

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Beyond the bench: Revitalizing ataluren development for rare genetic disorders 超越替补席：重振罕见遗传疾病的人才发展

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-21 DOI: 10.1016/j.ymthe.2025.06.002

Valentino Bezzerri, Marco Cipolli

引用次数: 0

Novel AAV-based GJB2 gene therapy restores hearing function 新的基于aav的GJB2基因治疗恢复听力功能

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-21 DOI: 10.1016/j.ymthe.2025.06.006

Lukas D. Landegger

引用次数: 0

Targeting EBV gp42 for nasopharyngeal carcinoma prevention 靶向EBV gp42预防鼻咽癌

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-21 DOI: 10.1016/j.ymthe.2025.06.005

Cong Sun, Le-Le Zhang, Mu-Sheng Zeng

引用次数: 0

Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche 利用b19导向的CAR - T细胞在血清阳性患者中进行AAV载体管理：生态位的重要性

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-21 DOI: 10.1016/j.ymthe.2025.06.007

Hanadi Saliba, David Alexandre Gross, Giuseppe Ronzitti

引用次数: 0

Bispecific receptor decoys: A new player to a crowded field 双特异性受体诱饵：一个进入拥挤区域的新玩家

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-21 DOI: 10.1016/j.ymthe.2025.06.015

Glenn Yiu

引用次数: 0

Retraction Notice to: LncRNA ODRUL Contributes to Osteosarcoma Progression through the miR-3182/MMP2 Axis. 撤回通知：LncRNA ODRUL通过miR-3182/MMP2轴促进骨肉瘤进展。

IF 12.1 1区医学

Molecular Therapy Pub Date : 2025-06-19 DOI: 10.1016/j.ymthe.2025.06.030

Kun-Peng Zhu, Xiao-Long Ma, Chun-Lin Zhang

引用次数: 0

Effective Use of BCMA-Targeting Bispecific T-Cell-Engaging Antibody in Treatment Refractory LRP4-positive Myasthenia Gravis. 靶向bcma的双特异性t细胞结合抗体在治疗难治性lrp4阳性重症肌无力中的有效应用

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-17 DOI: 10.1016/j.ymthe.2025.06.029

Stefanie Schreiber,Marwa Al-Dubai,Stefan Vielhaber,Lora Lefterova,Sascha Dietrich,Tobias Ruck,Sven Meuth,Denise Walther,Dimitrios Mougiakakos

{"title":"Effective Use of BCMA-Targeting Bispecific T-Cell-Engaging Antibody in Treatment Refractory LRP4-positive Myasthenia Gravis.","authors":"Stefanie Schreiber,Marwa Al-Dubai,Stefan Vielhaber,Lora Lefterova,Sascha Dietrich,Tobias Ruck,Sven Meuth,Denise Walther,Dimitrios Mougiakakos","doi":"10.1016/j.ymthe.2025.06.029","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.06.029","url":null,"abstract":"Myasthenia gravis (MG) is an antibody-mediated autoimmune disease affecting the neuromuscular junction. Refractory MG, particularly in cases associated with rare anti-LRP4 antibodies, presents significant treatment challenges. Teclistamab, a bispecific antibody targeting BCMA and CD3, redirects T cells against plasma cells and is approved for multiple myeloma treatment. Increasing interest surrounds the use of T-cell-based therapies in B-cell-mediated autoimmune diseases, particularly CAR T cells, which have demonstrated efficacy in achieving deep B-cell depletion and durable remissions. Additionally, emerging data support the use of T cell engagers (TCEs) as an alternative strategy for targeting autoreactive B cells. We report the case of a 47-year-old woman with severe, refractory, LRP4-positive MG. Despite multiple treatments, she remained severely affected, wheelchair-bound, and experiencing significant disability. Off-label teclistamab administration resulted in mild cytokine release syndrome (CRS), self-resolving lymphopenia, and hypogammaglobulinemia. Residual circulating B cells were eliminated, anti-LRP4 antibodies became undetectable, and clinical scores improved significantly. The patient regained mobility and has sustained remission during short-term follow-up. This case highlights the therapeutic potential of teclistamab and BCMA-targeting strategies in MG, warranting further investigation.","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"44 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144320095","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}

引用次数: 0

CRISPR for Cystic Fibrosis: advances and insights from a systematic review. CRISPR治疗囊性纤维化：来自系统综述的进展和见解。

IF 12.4 1区医学

Molecular Therapy Pub Date : 2025-06-17 DOI: 10.1016/j.ymthe.2025.06.021

Lucia Nicosia,Patrick T Harrison

{"title":"CRISPR for Cystic Fibrosis: advances and insights from a systematic review.","authors":"Lucia Nicosia,Patrick T Harrison","doi":"10.1016/j.ymthe.2025.06.021","DOIUrl":"https://doi.org/10.1016/j.ymthe.2025.06.021","url":null,"abstract":"Cystic Fibrosis (CF) is a severe genetic disorder caused by loss-of-function mutations in the CFTR gene. Gene editing approaches have the potential to correct such mutations. This systematic review outlines the mechanisms of the main CRISPR-based technologies, and, through cross-study comparisons, analyzes twenty-seven research articles that applied these technologies to target CF-causing variants. We report and discuss the strategy design, target cell selection, editing efficiency, prevalence of editing byproducts and levels of CFTR functional restoration achieved in each work, with the aim of providing technical insights for further exploration of CRISPR-based gene editing approaches. Our findings show that the F508del and W1282X mutations were the most extensively studied CF-causing variants, though over fifteen mutations were targeted overall. The majority of works under review explored the use of homology-directed repair (HDR) or base editing, with a growing number reporting efficient prime editing. Some studies tackled multiple individual mutations, compared different editors, or tested strategies across various models, while others focused on approaches that rescue CFTR function without directly correcting a mutation. Several works also proposed strategies that could address multiple variants with a single approach, while others highlighted technical difficulties in editing certain regions of the CFTR gene. This cross-study comparison also emphasizes the need for standardized reporting of editing efficiency and functional rescue, and stresses the importance of further single-cell RNA sequencing and in-vivo studies to reach clinically-relevant conclusions. However, as gene editing techniques continue to evolve, and with over sixty ongoing CRISPR-based clinical trials, there is growing optimism for meaningful advancements in CF gene-editing therapeutics.","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":"89 1","pages":""},"PeriodicalIF":12.4,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144320097","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}

引用次数: 0