Gene Therapy

  • Methods Matter: Standard production platforms for recombinant AAV produce chemically and functionally distinct vectors

    Methods Matter: Standard production platforms for recombinant AAV produce chemically and functionally distinct vectors

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  • Quantitation of trace levels of DNA released from disrupted Adeno-associated virus gene therapy vectors

    Quantitation of trace levels of DNA released from disrupted Adeno-associated virus gene therapy vectors

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  • The importance of molecular interactions and stability in Gene Therapy46:44

    The importance of molecular interactions and stability in Gene Therapy

    New advances in gene therapy have triggered a resurgence of interest in the field. Though gene therapy offers exciting potential for the treatment of both monogenic disorders as well as more genetical

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  • Trying to develop safe and effective gene therapies?

    Biophysical methods can help.

    Find out how
  • Practical strategies for overcoming challenges in the development of AAV vectors for gene therapy38:40

    Practical strategies for overcoming challenges in the development of AAV vectors for gene therapy

    Gene therapy promises to treat and potentially cure a disease by correcting its underlying genetic cause. While gene therapies that utilize AAV vectors have shown encouraging results —including a few

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  • Solutions for gene therapy using a deeper analytical characterization of AAV vector development18:38

    Solutions for gene therapy using a deeper analytical characterization of AAV vector development

    Gene therapy—a type of DNA-based medicine that utilizes a virus to insert a healthy gene into cells to replace a mutated, disease-causing variant has emerged as one of the most promising medical treat

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  • Francisella novicida Cas9 interrogates genomic DNA with very high specificity and can be used for mammalian genome editing

    Francisella novicida Cas9 interrogates genomic DNA with very high specificity and can be used for mammalian genome editing

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  • Antibody-targeted chromatin enables effective intracellular delivery and functionality of CRISPR/Cas9 expression plasmids

    Antibody-targeted chromatin enables effective intracellular delivery and functionality of CRISPR/Cas9 expression plasmids

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  • Intrinsic differential scanning fluorimetry for fast and easy identification of Adeno-Associated Virus serotypes

    Intrinsic differential scanning fluorimetry for fast and easy identification of Adeno-Associated Virus serotypes

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  • Humanized Selective CD19CAR-T Cells Treatment Shows Efficacy in B-ALL Patients Who Relapsed after Receiving Murine-Based CD19CAR-T Therapies

    Humanized Selective CD19CAR-T Cells Treatment Shows Efficacy in B-ALL Patients Who Relapsed after Receiving Murine-Based CD19CAR-T Therapies

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  • Spatial screening of hemagglutinin on Influenza A Virus particles: Sialyl-LacNAc displays on DNA and PEG scaffolds reveal requirements

    Spatial screening of hemagglutinin on Influenza A Virus particles: Sialyl-LacNAc displays on DNA and PEG scaffolds reveal requirements

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