Nature biotechnology最新文献_第2页

As new cell and gene therapies emerge from academia, we must RISE to the opportunity

IF 33.1 1区生物学

Nature biotechnology Pub Date : 2025-01-17 DOI: 10.1038/s41587-024-02520-9

Nandhitha Uma Naresh, Dana Hammill, Mark Kessel, Roger J. Hajjar, Nathan L. Yozwiak

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Ionis wins FDA nod for triglyceride-lowering rare disease drug

IF 33.1 1区生物学

Nature biotechnology Pub Date : 2025-01-17 DOI: 10.1038/s41587-024-02546-z

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Pricey sickle cell gene therapies primed for change

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Nature biotechnology Pub Date : 2025-01-17 DOI: 10.1038/s41587-024-02542-3

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Therapeutic T cells with synthetic gene circuits act locally

IF 33.1 1区生物学

Nature biotechnology Pub Date : 2025-01-17 DOI: 10.1038/s41587-024-02541-4

Iris Marchal

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Cancer vaccines

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Nature biotechnology Pub Date : 2025-01-17 DOI: 10.1038/s41587-024-02521-8

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How will AI affect patent disclosures?

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Nature biotechnology Pub Date : 2025-01-17 DOI: 10.1038/s41587-024-02515-6

Lisa Larrimore Ouellette, Victoria Fang, Nicholas T. Ouellette

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Multiplexed inhibition of immunosuppressive genes with Cas13d for combinatorial cancer immunotherapy

IF 46.9 1区生物学

Nature biotechnology Pub Date : 2025-01-16 DOI: 10.1038/s41587-024-02535-2

Feifei Zhang, Ryan D. Chow, Emily He, Chuanpeng Dong, Shan Xin, Daniyal Mirza, Yanzhi Feng, Xiaolong Tian, Nipun Verma, Medha Majety, Yueqi Zhang, Guangchuan Wang, Sidi Chen

{"title":"Multiplexed inhibition of immunosuppressive genes with Cas13d for combinatorial cancer immunotherapy","authors":"Feifei Zhang, Ryan D. Chow, Emily He, Chuanpeng Dong, Shan Xin, Daniyal Mirza, Yanzhi Feng, Xiaolong Tian, Nipun Verma, Medha Majety, Yueqi Zhang, Guangchuan Wang, Sidi Chen","doi":"10.1038/s41587-024-02535-2","DOIUrl":"https://doi.org/10.1038/s41587-024-02535-2","url":null,"abstract":"The complex nature of the immunosuppressive tumor microenvironment (TME) requires multi-agent combinations for optimal immunotherapy. Here we describe multiplex universal combinatorial immunotherapy via gene silencing (MUCIG), which uses CRISPR–Cas13d to silence multiple endogenous immunosuppressive genes in the TME, promoting TME remodeling and enhancing antitumor immunity. MUCIG vectors targeting four genes delivered by adeno-associated virus (AAV) (Cd274/Pdl1, Lgals9/Galectin9, Lgals3/Galectin3 and Cd47; AAV-Cas13d-PGGC) demonstrate significant antitumor efficacy across multiple syngeneic tumor models, remodeling the TME by increasing CD8+ T-cell infiltration while reducing neutrophils. Whole transcriptome profiling validates the on-target knockdown of the four target genes and shows limited potential off-target or downstream gene alterations. AAV-Cas13d-PGGC outperforms corresponding shRNA treatments and individual gene knockdown. We further optimize MUCIG by employing high-fidelity Cas13d (hfCas13d), which similarly showed potent gene silencing and in vivo antitumor efficacy, without weight loss or liver toxicity. MUCIG represents a universal method to silence multiple immune genes in vivo in a programmable manner, offering broad efficacy across multiple tumor types.","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"20 1","pages":""},"PeriodicalIF":46.9,"publicationDate":"2025-01-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142986817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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Nanocarrier imaging at single-cell resolution across entire mouse bodies with deep learning

IF 46.9 1区生物学

Nature biotechnology Pub Date : 2025-01-14 DOI: 10.1038/s41587-024-02528-1

Jie Luo, Muge Molbay, Ying Chen, Izabela Horvath, Karoline Kadletz, Benjamin Kick, Shan Zhao, Rami Al-Maskari, Inderjeet Singh, Mayar Ali, Harsharan Singh Bhatia, David-Paul Minde, Moritz Negwer, Luciano Hoeher, Gian Marco Calandra, Bernhard Groschup, Jinpeng Su, Ceren Kimna, Zhouyi Rong, Nikolas Galensowske, Mihail Ivilinov Todorov, Denise Jeridi, Tzu-Lun Ohn, Stefan Roth, Alba Simats, Vikramjeet Singh, Igor Khalin, Chenchen Pan, Bernardo A. Arús, Oliver T. Bruns, Reinhard Zeidler, Arthur Liesz, Ulrike Protzer, Nikolaus Plesnila, Siegfried Ussar, Farida Hellal, Johannes Paetzold, Markus Elsner, Hendrik Dietz, Ali Erturk

{"title":"Nanocarrier imaging at single-cell resolution across entire mouse bodies with deep learning","authors":"Jie Luo, Muge Molbay, Ying Chen, Izabela Horvath, Karoline Kadletz, Benjamin Kick, Shan Zhao, Rami Al-Maskari, Inderjeet Singh, Mayar Ali, Harsharan Singh Bhatia, David-Paul Minde, Moritz Negwer, Luciano Hoeher, Gian Marco Calandra, Bernhard Groschup, Jinpeng Su, Ceren Kimna, Zhouyi Rong, Nikolas Galensowske, Mihail Ivilinov Todorov, Denise Jeridi, Tzu-Lun Ohn, Stefan Roth, Alba Simats, Vikramjeet Singh, Igor Khalin, Chenchen Pan, Bernardo A. Arús, Oliver T. Bruns, Reinhard Zeidler, Arthur Liesz, Ulrike Protzer, Nikolaus Plesnila, Siegfried Ussar, Farida Hellal, Johannes Paetzold, Markus Elsner, Hendrik Dietz, Ali Erturk","doi":"10.1038/s41587-024-02528-1","DOIUrl":"https://doi.org/10.1038/s41587-024-02528-1","url":null,"abstract":"Efficient and accurate nanocarrier development for targeted drug delivery is hindered by a lack of methods to analyze its cell-level biodistribution across whole organisms. Here we present Single Cell Precision Nanocarrier Identification (SCP-Nano), an integrated experimental and deep learning pipeline to comprehensively quantify the targeting of nanocarriers throughout the whole mouse body at single-cell resolution. SCP-Nano reveals the tissue distribution patterns of lipid nanoparticles (LNPs) after different injection routes at doses as low as 0.0005 mg kg−1—far below the detection limits of conventional whole body imaging techniques. We demonstrate that intramuscularly injected LNPs carrying SARS-CoV-2 spike mRNA reach heart tissue, leading to proteome changes, suggesting immune activation and blood vessel damage. SCP-Nano generalizes to various types of nanocarriers, including liposomes, polyplexes, DNA origami and adeno-associated viruses (AAVs), revealing that an AAV2 variant transduces adipocytes throughout the body. SCP-Nano enables comprehensive three-dimensional mapping of nanocarrier distribution throughout mouse bodies with high sensitivity and should accelerate the development of precise and safe nanocarrier-based therapeutics.","PeriodicalId":19084,"journal":{"name":"Nature biotechnology","volume":"36 1","pages":""},"PeriodicalIF":46.9,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142974844","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

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Publisher Correction: Engineered platelets as targeted protein degraders and application to breast cancer models

IF 46.9 1区生物学

Nature biotechnology Pub Date : 2025-01-13 DOI: 10.1038/s41587-025-02559-2

Yu Chen, Samira Pal, Wen Li, Fengyuan Liu, Sichen Yuan, Quanyin Hu

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Agriculture to flourish on precision breeding: who will benefit?

IF 33.1 1区生物学

Nature biotechnology Pub Date : 2025-01-13 DOI: 10.1038/s41587-024-02532-5

Cormac Sheridan

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