GenScript offers full range of services and products for Gene Therapy. From target discovery to lead identification and optimization to pre-clinical and clinical. We can support Gene Therapy companies throughout the whole process with our RUO, pre-clinical and clinical grade services and products.

Workflow Of Gene Therapy Development

Understanding of Disease Mechanism:

New insights into a disease process can be gained by bioinformatics approaches utilizing genomic information associated with the disease. A valuable tool for identifying the target genes involved in specific diseases can be the Whole Genome Screening using CRISPR gRNA Library. Next-generation sequencing and qPCR are other frequently utilized tools.

Target Gene Validation:

To validate targets, researchers typically use techniques such as cell-based assays, phenotypic models, IHC, FACS, WB, for target engagement to demonstrate that target is involved in the progression of a given disease.

Gene Therapy Resources

Explore our technologies and learn more about Gene Therapy . Find the information and resources you need by browsing through our educational assets.

  • Webinars: Target Discovery
    Unleash the power of NGS target enrichment by semiconductor

    Josh Wang, Ph.D.

    R&D Sr. Scientist Genscript USA Inc.


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    Building DNA Libraries to Explore the Combinatorial Design Space

    Yifan Li

    Senior scientist at GenScript USA Inc


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    CRISPR in Creating Knockin Cell Lines and Animal Models - Functionalizing Genome Editing for a Broad Range of Targets

    Shondra M. Pruett-Miller, PhD.

    Director, St. Jude Children’s Research Hospital Comprehensive Cancer Center


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    Effective gene editing using the CRISPR RNP system:higher editing efficiency, lower off-target effects, and minimal cytotoxicity

    James Chon, Ph.D.

    Field Application Scientist Genscript USA Inc.


    Watch Now
  • Webinars: Lead Identification and Optimization
    Library-selected AAV variants can effectively translate to non-human primates in the spinal cord and cochlea

    Killian S. Hanlon, PhD.

    Research Fellow, Harvard Medical School, Massachusetts General Hospital


    Watch Now
    Discovering the Next Generation AAV Vector Through Capsid Engineering and Expression Cassette Optimization

    Ye Liu, PhD.

    Senior Director of Gene Transfer Technologies, REGENXBIO Inc.


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    Solutions for in vivo barriers to gene therapy vectors

    Casey Maguire, PhD.

    Associate Professor of Neurology, Harvard Medical School and Investigator, Massachusetts General Hospita


    Watch Now
    Tailor-made mutant libraries for precision engineering

    Dr. Maung Nyan Win

    Sr. Application Scientist GenScript


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    Therapeutic Gene Editing Enabled by New Delivery Vehicles

    Niren Murthy, PhD.

    Professor, Department of Bioengineering UC Berkeley- innovative Genomics Institute


    Watch Now
  • Webinars: Pre-clinical Development
    Pre-clinical AAV production and optimization: Not as easy as it looks!

    Dr. Stephen Hughes

    Director of New Platform Development, GenScript


    Watch Now
    Precise and Efficient Non-viral CRISPR Gene Editing Solutions

    Lumeng Ye, Ph.D.

    Sr. Scientist, GenScript USA Inc.


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  • Webinars: Clinical Study
    Development and manufacturing Strategy for DNA Base Vaccine-Drug Candidates

    Mark Wei, Sr.

    Director of Plasmid & Virus Process Development & Manufacturing Department


    Watch Now
    Scalable AAV Manufacturing Strategies

    Dr. Xiao Pan

    Director of GenScript ProBio, Platform R&D Department


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    How to improve plasmid and viral vector manufacturing process

    Shigang Wei

    Director of Plasmid & Virus Process Development Department


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    Lentiviral vector manufacturing using suspension cell line

    Dr. Mahmood Ali

    Project Manager of Virus (R&D) Purification


    Watch Now
  • Technical Resource Center
  • Supplement Your Research With Our Technical Resources!
  • Bioinformatics Tools
  • Learning Center
  • Here you can explore our free educational resources to help you understand the latest technologies supporting life science research, and learn about using GenScript's custom services and products to accelerate your research.

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