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Nature Biotechnology

Resumen/Descripción – provisto por la editorial en inglés
Nature Biotechnology is a monthly journal covering the science and business of biotechnology. It publishes new concepts in technology/methodology of relevance to the biological, biomedical, agricultural and environmental sciences as well as covers the commercial, political, ethical, legal, and societal aspects of this research. The first function is fulfilled by the peer-reviewed research section, the second by the expository efforts in the front of the journal. We provide researchers with news about business; we provide the business community with news about research developments.
Palabras clave – provistas por la editorial

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Institución detectada Período Navegá Descargá Solicitá
No detectada desde jul. 2012 / hasta dic. 2023 Nature.com

Información

Tipo de recurso:

revistas

ISSN impreso

1087-0156

ISSN electrónico

1546-1696

Editor responsable

Springer Nature

País de edición

Reino Unido

Fecha de publicación

Tabla de contenidos

Programmable protein expression using a genetically encoded m6A sensor

Bahjat F. Marayati; Matthew G. ThompsonORCID; Christopher L. HolleyORCID; Stacy M. HornerORCID; Kate D. MeyerORCID

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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GEMS enables mRNA m6A methylation sensing in living cells and m6A-coupled protein delivery

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Metabolically enhanced CAR T cells efficiently clear solid tumors in mice

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Combined small-molecule treatment accelerates maturation of human pluripotent stem cell-derived neurons

Emiliano Hergenreder; Andrew P. Minotti; Yana Zorina; Polina Oberst; Zeping Zhao; Hermany MungubaORCID; Elizabeth L. Calder; Arianna BaggioliniORCID; Ryan M. Walsh; Conor ListonORCID; Joshua Levitz; Ralph Garippa; Shuibing ChenORCID; Gabriele CiceriORCID; Lorenz StuderORCID

<jats:title>Abstract</jats:title><jats:p>The maturation of human pluripotent stem cell (hPSC)-derived neurons mimics the protracted timing of human brain development, extending over months to years for reaching adult-like function. Prolonged in vitro maturation presents a major challenge to stem cell-based applications in modeling and treating neurological disease. Therefore, we designed a high-content imaging assay based on morphological and functional readouts in hPSC-derived cortical neurons which identified multiple compounds that drive neuronal maturation including inhibitors of lysine-specific demethylase 1 and disruptor of telomerase-like 1 and activators of calcium-dependent transcription. A cocktail of four factors, GSK2879552, EPZ-5676, <jats:italic>N</jats:italic>-methyl-<jats:sc>d</jats:sc>-aspartate and Bay K 8644, collectively termed GENtoniK, triggered maturation across all parameters tested, including synaptic density, electrophysiology and transcriptomics. Maturation effects were further validated in cortical organoids, spinal motoneurons and non-neural lineages including melanocytes and pancreatic β-cells. The effects on maturation observed across a broad range of hPSC-derived cell types indicate that some of the mechanisms controlling the timing of human maturation might be shared across lineages.</jats:p>

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Large-scale evaluation of the ability of RNA-binding proteins to activate exon inclusion

Jonathan C. SchmokORCID; Manya Jain; Lena A. StreetORCID; Alex T. Tankka; Danielle Schafer; Hsuan-Lin Her; Sara Elmsaouri; Maya L. GosztylaORCID; Evan A. Boyle; Pratibha Jagannatha; En-Ching Luo; Ester J. Kwon; Marko Jovanovic; Gene W. YeoORCID

<jats:title>Abstract</jats:title><jats:p>RNA-binding proteins (RBPs) modulate alternative splicing outcomes to determine isoform expression and cellular survival. To identify RBPs that directly drive alternative exon inclusion, we developed tethered function luciferase-based splicing reporters that provide rapid, scalable and robust readouts of exon inclusion changes and used these to evaluate 718 human RBPs. We performed enhanced cross-linking immunoprecipitation, RNA sequencing and affinity purification–mass spectrometry to investigate a subset of candidates with no prior association with splicing. Integrative analysis of these assays indicates surprising roles for TRNAU1AP, SCAF8 and RTCA in the modulation of hundreds of endogenous splicing events. We also leveraged our tethering assays and top candidates to identify potent and compact exon inclusion activation domains for splicing modulation applications. Using these identified domains, we engineered programmable fusion proteins that outperform current artificial splicing factors at manipulating inclusion of reporter and endogenous exons. This tethering approach characterizes the ability of RBPs to induce exon inclusion and yields new molecular parts for programmable splicing control.</jats:p>

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Identification of a chemical cocktail that drives the maturation of human neurons

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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A step closer to safe and efficient mammalian gene regulation

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Discovery of sparse, reliable omic biomarkers with Stabl

Julien Hédou; Ivana MarićORCID; Grégoire BellanORCID; Jakob Einhaus; Dyani K. GaudillièreORCID; Francois-Xavier Ladant; Franck VerdonkORCID; Ina A. StelzerORCID; Dorien FeyaertsORCID; Amy S. TsaiORCID; Edward A. Ganio; Maximilian Sabayev; Joshua Gillard; Jonas Amar; Amelie Cambriel; Tomiko T. Oskotsky; Alennie RoldanORCID; Jonathan L. Golob; Marina SirotaORCID; Thomas A. Bonham; Masaki SatoORCID; Maïgane Diop; Xavier Durand; Martin S. AngstORCID; David K. Stevenson; Nima AghaeepourORCID; Andrea Montanari; Brice GaudillièreORCID

<jats:title>Abstract</jats:title><jats:p>Adoption of high-content omic technologies in clinical studies, coupled with computational methods, has yielded an abundance of candidate biomarkers. However, translating such findings into bona fide clinical biomarkers remains challenging. To facilitate this process, we introduce Stabl, a general machine learning method that identifies a sparse, reliable set of biomarkers by integrating noise injection and a data-driven signal-to-noise threshold into multivariable predictive modeling. Evaluation of Stabl on synthetic datasets and five independent clinical studies demonstrates improved biomarker sparsity and reliability compared to commonly used sparsity-promoting regularization methods while maintaining predictive performance; it distills datasets containing 1,400–35,000 features down to 4–34 candidate biomarkers. Stabl extends to multi-omic integration tasks, enabling biological interpretation of complex predictive models, as it hones in on a shortlist of proteomic, metabolomic and cytometric events predicting labor onset, microbial biomarkers of pre-term birth and a pre-operative immune signature of post-surgical infections. Stabl is available at <jats:ext-link xmlns:xlink="http://www.w3.org/1999/xlink" ext-link-type="uri" xlink:href="https://github.com/gregbellan/Stabl">https://github.com/gregbellan/Stabl</jats:ext-link>.</jats:p>

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Cell and gene therapy approvals in Japan and the need for international harmonization

Teruyo AratoORCID; Kaori NomuraORCID

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo

Meirui AnORCID; Aditya Raguram; Samuel W. DuORCID; Samagya Banskota; Jessie R. Davis; Gregory A. Newby; Paul Z. ChenORCID; Krzysztof PalczewskiORCID; David R. LiuORCID

<jats:title>Abstract</jats:title><jats:p>Prime editing enables precise installation of genomic substitutions, insertions and deletions in living systems. Efficient in vitro and in vivo delivery of prime editing components, however, remains a challenge. Here we report prime editor engineered virus-like particles (PE-eVLPs) that deliver prime editor proteins, prime editing guide RNAs and nicking single guide RNAs as transient ribonucleoprotein complexes. We systematically engineered v3 and v3b PE-eVLPs with 65- to 170-fold higher editing efficiency in human cells compared to a PE-eVLP construct based on our previously reported base editor eVLP architecture. In two mouse models of genetic blindness, single injections of v3 PE-eVLPs resulted in therapeutically relevant levels of prime editing in the retina, protein expression restoration and partial visual function rescue. Optimized PE-eVLPs support transient in vivo delivery of prime editor ribonucleoproteins, enhancing the potential safety of prime editing by reducing off-target editing and obviating the possibility of oncogenic transgene integration.</jats:p>

Palabras clave: Biomedical Engineering; Molecular Medicine; Applied Microbiology and Biotechnology; Bioengineering; Biotechnology.

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