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Evolution in gene therapy vector analysis

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Discover the latest technology to supercharge your gene therapy vector analysis workflow

16 Apr 2024
Lawrence Howes
Editorial Assistant

Editorial article

Eileen Hannigan and Sarah Nadin, Assay Development team at Merck
Viral vectors are pivotal for delivering functional genes to individuals with genetic disorders

Within the ever-evolving field of gene therapy, vectors play an indispensable role as essential carriers, delivering therapeutic genes into target cells. Viral vectors, such as the adeno-associated virus vector (AAV), are commonly employed for this purpose. These genetic vehicles are pivotal for delivering functional genes to individuals with genetic disorders and therefore require rigorous quality analysis.

Vector analysis involves the assessment of critical quality attributes such as structural integrity, genetic payload accuracy, strength, and purity. Techniques such as PCR, next-generation sequencing, and mass spectrometry are deployed for this purpose, shedding light on the vectors' safety, effectiveness, and overall success in the realm of gene therapy.

This top list has been curated by our expert editors at SelectScience® and encompasses a range of what we believe to be some of the most exciting developments in gene therapy vector analysis.

1. Rapid characterization of AAV critical product quality attributes

908 Devices Inc. presents its ZipChip™ CE-MS device, a revolutionary tool for rapidly characterizing critical product quality attributes in biologics, oligonucleotides, RNAs, and AAVs. The company is transforming chemical and biochemical analysis through user-friendly mass spectrometry devices that deliver rapid results on-site. The ZipChip device utilizes microchip-based capillary electrophoresis (CE) and nano-electrospray ionization technology, streamlining sample preparation, and enabling high-speed and high-resolution separations.

2. Boost viral vector production with chemically defined viral feeds

Discover FUJIFILM Irvine Scientific’s latest chemically defined, nutrient-based HEK293-specific feed medium, designed to boost AAV production for gene therapy applications and viral vector-based vaccines.

3. Tissue biodistribution of AAV-based gene therapy

AAV tissue biodistribution and cell tropism are essential pieces of information for every gene therapy development program. Download this application note to learn about the RNAscope® ISH assay, a highly informative method for quantitative, cell-specific measurement of AAV vector and transgene expression.

4. Automation for viral titer and impurity analyses

Immunoassays are used for vector titer and impurity analysis. However, limitations in current methods can create bottlenecks in workflows, and the development of high-quality assays for biotherapeutic development can be difficult and time-consuming. Learn how automated platforms such as the Gyrolab® Immunoassay systems from Gyros Protein Technologies can improve the efficiency of assay-based processes and can support the development of custom assays.

5. Optimize osmolality to improve AAV production

The use of viral vectors is now a common approach within cell and gene therapy, but there are still difficulties when scaling up for bioprocessing. This study, presented by Advanced Instruments and undertaken by Cell and Gene Therapy Catapult, provides preliminary evidence for impact on overall yield, and quality of the adeno-associated virus 1 (AAV) due to a timed osmolality shift with and without a feeding step.

6. Solutions to AAV-based gene therapy manufacturing challenges

In this article, Dr. Markus Krieger, Vice President, Technology Development, and Clive Glover, Viral Vector Leader at Cytiva, discuss the challenges that the emerging field of adeno-associated virus (AAV)-based therapies is facing. They also share their thoughts on the methods used to manufacture AAVs and how co-optimizing components in the viral vector workflow may help solve a lot of the hurdles in the field today.

7. AAV manufacturing workflow

Download this application note by Cytiva to learn about the objectives, considerations, and strategies for each step of an AAV manufacturing workflow. Additionally discover the biotherapeutic characterization technology designed to streamline the determination of potency, purity, and strength of your AAV products.

8. Sharpen your gene therapy toolkit with static light scattering (SLS)

NanoTemper Technologies has now added static light scattering (SLS) to its Prometheus Panta, allowing scientists to characterize their AAV vectors – whether it be serotyping, formulation, or capsid filling and leaking – with very little sample. Read more about the Prometheus Panta SLS here.

Discover the latest news on gene therapy by signing up for our free newswires, where our expert editors collate the latest products and news in the field.

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Genome AnalysisGenomics, the study of genomes, includes functional genomics, evolutionary genomics and comparative genomics. There are many genomic technologies such as DNA sequencing of whole genomes, computational biology and bioinformatics. DNA and nucleic acids must be isolated and concentrated from cells for analysis with kits, automated analyzers and software. Other useful technologies for studying genomics include PCR, microarrays and electrophoresis.ProteomicsProteomics is the systemic bioinformatics study of proteins and amino acids, including their structure, size, function and identification. Tools used in proteomics include chromatography, blotting and gels, protein arrays, mass spectrometry and ELISA and associated analysis software. Analyzers and proteomic systems should be sensitive, high resolution, fast and may be automated for high-throughput.PCR and Thermal CyclingPolymerase chain reaction (PCR) kits and thermal cyclers are used for the in vitro amplification of DNA permitting subsequent analysis and experimental procedures. Explore a range of high-quality polymerase, primers and nucleotides or simplify your workflow with a PCR mastermix. Find reverse transcription PCR (RT-PCR) and cDNA synthesis kits for RNA products and libraries. Quantitatively measure the amplification of DNA with real-time PCR (qPCR) and droplet digital PCR (ddPCR) kits and systems, and discover automated PCR setup solutions to increase throughput. Alternative DNA amplification methods also include recombinase polymerase amplification (RPA) kits. Find the best PCR kits and thermal cyclers and purification equipment in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Cell Lines Stem Cells and Primary CellsPrimary cell cultures, established cell lines and stem cells are vital for <i>in vitro</i> and <i>ex vivo</i> experimentation. High-quality cells, optimized for your applications, alongside optimized cell substrates, growth medium and supplements, are critical for experimental success. Explore a range of cells suitable for your applications, including isogenic cell lines, competent cells, induced pluripotent stem cell (iPSC)-derived cell lines, fungal/bacterial/mammalian cell lines, stem cells and cancer cell lines. Find the best cells for your research in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.DNA / RNA Extraction and PurificationPurified DNA and RNA are required for numerous downstream molecular biology applications. Consequently, the importance of high-quality DNA/RNA extraction and purification equipment cannot be underestimated. Many purification kits are available and are typically optimized for nucleic acid type and source, including plasmid DNA, genomic DNA, mRNA, RNA and viral nucleic acid purification kits. Automated extraction and purification of nucleic acids can be implemented with magnetic bead separator instruments or high-throughput purification workstations. Find the best DNA/RNA extraction and purification equipment in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.DNA SequencingDNA sequencing, such as sanger sequencing, is a biological technique that determines the precise order of nucleotide bases in a fragment or template of DNA. DNA sequencers and genetic analyzers are based on capillary electrophoresis, where labeled DNA fragments are electrophoretically separated by size as they migrate through a polymer. Find the best DNA sequencing products, including DNA sequencing kits, genomic libraries and genetic identity kits in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Protein PurificationProtein purification is a vital step in drug discovery, therapeutics, biotech and life science research. The purification process typically involves subcellular or membrane protein extraction with cell lysis kits, separation of proteins from cell debris by filtration or spin columns, and the isolation of proteins of interest from other proteins and impurities with affinity purification (including fusion protein tags and antibody binding proteins A, G and L), immunoprecipitation or chromatographic methods, such as ion exchange, size exclusion and immobilized metal affinity chromatography. All purification methods come in multiple formats for your laboratory needs, including agarose or magnetic beads, resins, columns and filter plates. Find the best protein purification equipment in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Gene Expression and Molecular CloningMolecular cloning is a set of techniques that utilizes vectors to transfer recombinant DNA into host cells and is an essential tool for investigating the expression of genes and proteins in bacterial or mammalian cells. A variety of vectors optimized for gene cloning and expression in a range of host organisms are available, alongside competent cells for genetic replication. Here, you can explore a range of molecular tools, high-quality genomic and cDNA libraries, premade clones, transformation and transfection reagents and mutagenesis or gene expression detection assays and expression arrays. Find the best gene expression and molecular cloning products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Next Generation SequencingNext-generation sequencing (NGS), also known as whole-genome sequencing, high-throughput sequencing and massive parallel sequencing, produces and analyses thousands to millions of nucleotide sequences at once. Sequencing systems operate via varying technologies depending on the manufacturer, including sequencing by synthesis, ligation, pyrosequencing, ion semiconductor and single-molecule real-time sequencing. For NGS, library preparation is paramount to successful sequencing. In this section, explore a range of library preparation kits, from targeted, amplicon-based or hybridization-based kits including epigenomic, transcriptomic and genomic workflows to fragmentation kits. Find the best next-generation sequencing products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Protein QuantificationThe detection and quantification of proteins in a sample is vital across life sciences, pharmaceutical research and clinical diagnostics, and a variety of equipment is available to scientists to simplify the workflow. Proteins of interest can be easily labeled and detected on light-based detection instruments. Immunoassay kits allow you to identify a specific protein of interest and protein detection beads or antibody microarrays allow you to identify multiple specific proteins at once. Protein interactions and enzyme activity can also be monitored with protein-protein interaction assays. Additionally, the biophysical characterization of proteins is made easy with biokinetic analyzers. Find the best protein detection and quantification products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.DNA / RNA QuantificationDetection and quantification of nucleic acids is important in molecular biology, cloning, expression, forensics and clinical diagnostics. Nucleic acids can be detected by labeling with colorimetric, fluorescent or radio labels and using in situ hybridization kits to identify specific sequences. Multiple nucleic acids can be detected and quantified at once using RNA / DNA detection beads or RNA / DNA microarrays. Find the best DNA / RNA Quantification products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Protein ExpressionProtein expression is the utilization of cell machinery for the synthesis of proteins and has become a critical tool in biotherapeutic, genomic, and proteomic research. Produce recombinant proteins with expression vectors in combination with a host cell suitable for high-level protein expression. For production of toxic proteins, consider cell-free expression vectors. Create and monitor post-translational modifications with protein modification kits. Find the best protein expression products in our peer-reviewed product directory: compare products, check customer reviews and receive pricing direct from manufacturers.Clinical GeneticsMolecular Genetics covers the analysis of hereditary genetic disease and chromosomal abnormalities. Genetics can be analysed using DNA, RNA, and protein microarrays, PCR, RT PCR and DNA sequencing. Genetic equipment includes genetic workstations, thermal cyclers, cooling blocks and electrophoresis products. Diagnostic kits are used for DNA / RNA extraction and purification.Gene EditingGene editing is the precise alteration to an organism's DNA, with CRISPR being the most well-known technique. It is used in a wide range of applications, including the development of disease models and gene therapy, and crop improvement. Browse our peer-reviewed product directory to find the best gene editing solutions, compare products, check reviews, and get pricing directly from manufacturers.Gene TherapyGene therapy is a technique that modifies or replaces genes within an individual's cells to treat or prevent diseases. This approach holds potential for curing genetic disorders, cancers, and certain viral infections. Advances in gene editing technologies like CRISPR are driving the growth of gene therapy. Explore gene therapy tools and technologies in our peer-reviewed product directory; compare products, check reviews, and get pricing directly from manufacturers.

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