Search Results - "De Alencastro, Gustavo"

The Transcriptional and Functional Properties of Mouse Epiblast Stem Cells Resemble the Anterior Primitive Streak by Kojima, Yoji, Kaufman-Francis, Keren, Studdert, Joshua B., Steiner, Kirsten A., Power, Melinda D., Loebel, David A.F., Jones, Vanessa, Hor, Angelyn, de Alencastro, Gustavo, Logan, Grant J., Teber, Erdahl T., Tam, Oliver H., Stutz, Michael D., Alexander, Ian E., Pickett, Hilda A., Tam, Patrick P.L.

“…Mouse epiblast stem cells (EpiSCs) can be derived from a wide range of developmental stages. To characterize and compare EpiSCs with different origins, we…”
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Improved Genome Editing through Inhibition of FANCM and Members of the BTR Dissolvase Complex by de Alencastro, Gustavo, Puzzo, Francesco, Pavel-Dinu, Mara, Zhang, Feijie, Pillay, Sirika, Majzoub, Karim, Tiffany, Matthew, Jang, Hagoon, Sheikali, Adam, Cromer, M. Kyle, Meetei, Ruhikanta, Carette, Jan E., Porteus, Matthew H., Pekrun, Katja, Kay, Mark A.

“…Recombinant adeno-associated virus (rAAV) vectors have the unique property of being able to perform genomic targeted integration (TI) without inducing a…”
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Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system by Cunningham, Sharon C., Siew, Susan M., Hallwirth, Claus V., Bolitho, Christine, Sasaki, Natsuki, Garg, Gagan, Michael, Iacovos P., Hetherington, Nicola A., Carpenter, Kevin, de Alencastro, Gustavo, Nagy, Andras, Alexander, Ian E.

“…Liver‐targeted gene therapy based on recombinant adeno‐associated viral vectors (rAAV) shows promising therapeutic efficacy in animal models and adult‐focused…”
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Collybistin and gephyrin are novel components of the eukaryotic translation initiation factor 3 complex by Sertie, Andrea L, de Alencastro, Gustavo, De Paula, Vanessa J, Passos-Bueno, Maria Rita

“…Collybistin (CB), a neuron-specific guanine nucleotide exchange factor, has been implicated in targeting gephyrin-GABAA receptors clusters to inhibitory…”
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Tracking Adeno-Associated Virus Capsid Evolution by High-Throughput Sequencing by de Alencastro, Gustavo, Pekrun, Katja, Valdmanis, Paul, Tiffany, Matthew, Xu, Jianpeng, Kay, Mark A

“…Despite early successes using recombinant adeno-associated virus (rAAV) vectors in clinical gene therapy trials, limitations remain making additional…”
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539. Screening for Recombinant Adeno-Associated Viral Vectors That Selectively Transduce Hepatitis B Virus Infected Cells by de Alencastro, Gustavo, Pekrun, Katja, Kay, Mark

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Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice by Tsuji, Shinnosuke, Stephens, Calvin J., Bortolussi, Giulia, Zhang, Feijie, Baj, Gabriele, Jang, Hagoon, de Alencastro, Gustavo, Muro, Andrés F., Pekrun, Katja, Kay, Mark A.

“…Homologous recombination (HR)-based gene therapy using adeno-associated viruses (AAV-HR) without nucleases has several advantages over classic gene therapy,…”
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Using a barcoded AAV capsid library to select for clinically relevant gene therapy vectors by Pekrun, Katja, De Alencastro, Gustavo, Luo, Qing-Jun, Liu, Jun, Kim, Youngjin, Nygaard, Sean, Galivo, Feorillo, Zhang, Feijie, Song, Ren, Tiffany, Matthew R, Xu, Jianpeng, Hebrok, Matthias, Grompe, Markus, Kay, Mark A

“…While gene transfer using recombinant adeno-associated viral (rAAV) vectors has shown success in some clinical trials, there remain many tissues that are not…”
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305. A Screening Strategy for Selecting Recombinant Adeno-Associated Viral Vectors That Selectively Transduce Viral Infected Cells by de Alencastro, Gustavo, Paulk, Nicole K., Pekrun, Katja, Kay, Mark A.

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Exploiting the unique regenerative capacity of the liver to underpin cell and gene therapy strategies for genetic and acquired liver disease by Logan, Grant J., de Alencastro, Gustavo, Alexander, Ian E., Yeoh, George C.

“…The number of genetic or acquired diseases of the liver treatable by organ transplantation is ever-increasing as transplantation techniques improve placing…”
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335. Toward a Cellular Therapy for Metabolic Liver Disease: Gene Targeting of the Ornithine Transcarbamylase (Otc) Locus in Bipotential Murine Oval Liver Cells (BMOL) Using Adeno-Associated Virus by Logan, Grant J., Woo, Ken H., Smyth, Christine M., Cunningham, Sharon C., Trippit, Jessica R., de Alencastro, Gustavo, Yeoh, George C.T., Alexander, Ian E.

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700. Stable Correction of Severe Metabolic Liver Disease Phenotypes in the Growing Murine Liver Using a Hybrid rAAV/piggyBac Transposon Gene Delivery System by Cunningham, Sharon C., Siew, Susan M., Hallwirth, Claus V., Bolitho, Christine, Sasaki, Natsuki, Garg, Gagan, Michael, Iacovos P., Hetherington, Nicola A., de Alencastro, Gustavo, Nagy, Andras, Alexander, Ian E.

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