What is Genome Editing and CRISPR?

Gene editing has garnered significant attention in recent years, but that may leave you wondering: what exactly is it? It is a form of one-time therapy that directly edits pieces of DNA within a cell.

Several approaches to genome editing have been developed. Before the advent of CRISPR-Cas9, two approaches were used to make site-specific double-stranded breaks in DNA: zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs).

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene editing technology that allows scientists to make precise changes to DNA.

CRISPR technology can be used to make DNA changes inside a cell. These cells can be edited inside the body (in vivo) or outside the body (ex vivo) from a patient or donor.

GenScript is simplifying gene editing with user-friendly online design tools, useful protocols, and expert technical support to help you harness the power of CRISPR genome editingfor your therapeutic development.

Ex vivo
Gene Editing
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In vivo
Gene Editing
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Ex vivo Gene Editing

Ex vivo CRISPR genome editing is a therapeutic approach in which the genome of cells are edited in vitro, and then those modified cells , such as T cells or HSCs, are transplanted back into the patient to produce a therapeutic resulting from the genome editing.

Service Recommendations:

  • Research Use Only sgRNA & Nucleases:

    EasyEdit sgRNA: Synthetic sgRNAs with modifications for reliable gene editing

    Most economical solution icon

    Most economical solution

    Ideal for early research & discovery
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    High editing efficiency

    Regardless of cell type
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    Flexible at scale

    1 to 1000+ sgRNAs 2 to 100+ nmol

    SafeEdit sgRNA: HPLC purified for improved performance

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    Minimal impact for
    cell viability

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    Reduced off-targeting from
    truncated guides

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    Ideal for primary cells
    & stem cells

    Cas9 / Cas12a / Cas13a Nucleases:

    For precise RNA-guided DNA or RNA editing and detection

    RNP-mediated CRISPR Gene Editing

    • Non-viral delivery, no risk of gene integration
    • Fast editing action and degradation
    • Low cytotoxicity and immunogenicity

    Consistent high knockout and knock-in efficiency

    • In tested cell lines and primary T cells
    • At different electroporation scales
    • Using all grades of Ultra Cas9 Nucleases

    High Lot-to-lot Consistency

    • In in vitro cleavage assay
    • In gene editing applications
  • Available HDR Formats
    GenExact™ ssDNA
    Precise KI and low cytotoxicity
    GenExact ssDNA

    Application

    Ideal for T cell therapy

    Ideal for cell/animal model generation

    Advantage

    Minimal cytotoxicity

    Precise knock-in, minimized off-target effects

    High purity and sequence-verified

    Specifications

    Insertion length: 150-5,000 nt

    µg to mg scale

    Research to cGMP grade

    GenWand™ dsDNA
    Long gene knock-in in large scale

    Application

    Ideal for long gene CRISPR KI

    Ideal for screening and scale-up

    Advantage

    Covalently closed ends protect for better accuracy

    More suitable for scale-up

    High purity and sequence-verified

    Specifications

    Insertion length: 2-10 kb

    µg to g scale

    Research to cGMP grade

    GenCircle™ dsDNA
    Circular template with no resistance gene

    Application

    Ideal replacement for plasmid HDR template

    Ideal for scale-up and fast delievery

    Advantage

    KI efficiency increase by up to 30%

    429bp backbone, lower cytotoxicity & higher transfection efficiency

    No antibiotic resistance avoids regulatory concern

    High purity and sequence-verified

    Specifications

    Insertion length: 1-50 kb

    µg to g scale

    cGMP grade coming soon

    gRNA & HDR Template Design Tools: makes guide RNA and HDR template design easy

    Start your project

  • GenCRISPR cGMP sgRNA
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    State-of-the-Art Production Facility

    • cGMP manufacturing of 30mg up to gram quantities per batch
    • Compliant with FDA/EMA/PMDA/NMPA regulations
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    Comprehensive QA/QC Documentation

    • 10+ QC options supporting global IND filing needs
    • US Type II DMF and/or CMC filing support
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    Trusted Partner from Bench to Clinic

    • Successfully delivered 100+ cGMP batches
    • 6 IND approvals
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    Production Facility
    cGMP manufacturing facility opened 2022

    cGMP production environment

    Clean suite with class A isolator in a class C background

    Sterile filtration and aseptic fill finish

    Process development and optimization

    CQA/CPP confirmed

    Flexible batch scale from 30 mg – 1 g

    QA/QC & Documentation Banner
    QA/QC & Documentation

    Manufacturing Summary Report, TSE/BSE Statement

    Batch Records based on Master Batch Record and Change Control System

    Identity, purity, sterility, etc. clearly defined

    cGMP-compliant material and supplier management

    Validated QC testing procedures

    Long-term stability testing under various solutions and storage conditions

    Established QMS for training, process deviations, and CAPA

    Specifications

    RUO GMP-like cGMP
    Quantity µg - mg mg - g mg - g
    Purification & Analysis Desalt or HPLC 90% NGS Full Report
    Analytical Methods Suitably Verified Qualified Validated
    Documentation COA Customizable Full GMP
    Facility Controlled, Unclassified Classified upon request Grade C+A (Isolator), capable of sterile product
    Materials Traceable with vendor management Traceable with vendor management Traceable with vendor management + ID testing
  • GenCRISPR cGMP HDR template
    Production Environment Banner
    Production Environment

    cGMP designated manufacturing lines/rooms

    Controlled production environment

    Cleaning process and cleaning records in place

    Qualified sterile filtration and aseptic processing fill/finish process

    Process Development Banner
    Process Development

    Process development and optimization

    Analytical test method development and optimization

    CQA/CPP Confirmation

    Quality Assurance & Documentation Banner
    Quality Assurance & Documentation

    Material management and supplier management in compliance with cGMP regulations

    Phase appropriate cGMP deployment based on stage of clinical trial and process understanding

    Basic Quality Management Systems in place (Training, Deviations, CAPA,...)

    Manufacturing Summary Report, TSE/BSE Statement

    Batch Records based on Master Batch Record and Change Control system

    Quality Control Banner
    Quality Control

    Identity, purity, sterility, etc. clearly defined

    Scientific sound QC test methods

    Stability Test Banner
    Stability Test

    Stability test under various solutions and storage conditions

    Prepare/initiate long term stability studies

    Specifications

    RUO GMP-like cGMP
    Analytical Methods Suitably Verified Qualified Validated
    Documentation CofA Customizable Full GMP
    Facility Controlled, Unclassified Classified upon request Grade C+A (isolator), capable for sterile product
    Materials Traceable with vendor management Traceable with vendor management Traceable with vendor management + ID testing
  • GMP Cas9 Nucleases

    Consistent High Editing Efficiency

    Consistent high knockout and knock-in efficiency

    • In tested cell lines and primary T cells
    • At different electroporation scale
    • Using different grade of Ultra Cas9 Nucleases

    High Lot-to-lot Consistency

    High lot-to-lot consistency

    • In in vitro cleavage assay
    • In gene editing applications

    Ultra High Stability

    Activity and purity maintain high and stable

    • In accelerated testing at 25°C and 4°C
    • In real-time stability testing at -20°C
    • In freeze-thaw stability testing

    Stringent Specifications

    Manufactured under guidelines for GMP, including

    • Aseptic manufacturing
    • Traceable documentation
    • Stringent testing

In vivo Gene Editing

In vivo CRISPR gene editing is a therapeutic approach in which the CRISPR components, such as Cas9 and guide RNA, are directly introduced into the patient’ body to edit the target genes. CRISPR components are delivered to specific target tissues or cells using viral vectors, nanoparticles, or other delivery systems. Inside the body, the CRISPR components can precisely edit target genes, correcting disease-causing mutations or regulating gene expression.

LNP-based delivery system

AAV-based delivery system 

Service Recommendations:

CRISPR sgRNA,HDR payloads and Cas Nucleases: Support from Research to Clinic

CRISPR sgRNA and HDR payloads : Support from Research to Clinic

GenScript Expands cGMP Capabilities for CRISPR sgRNA and payloads manufacturing

Gene Editing Resources

Explore our technologies and learn more about Gene Editing Solution. Find the information and resources you need by browsing through our educational material.

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

Watch Now
Solutions for in vivo barriers to gene therapy vectors
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
Therapeutic Gene Editing Enabled by New Delivery Vehicles
Therapeutic Gene Editing Enabled by New Delivery Vehicles

Niren Murthy, PhD
Professor, Department of Bioengineering UC Berkeley- innovative Genomics Institute

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

Lumeng Ye, Ph.D.
Sr. Scientist, GenScript USA Inc.

Watch Now
Pre-clinical AAV
                    production and optimization: Not as easy as it looks
Pre-clinical AAV production and optimization: Not as easy as it looks

Dr. Stephen Hughes
Director of New Platform Development, GenScript

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Scalable AAV Manufacturing Strategies
Scalable AAV Manufacturing Strategies

Dr. Xiao Pan
Director of GenScript ProBio, Platform R&D Department

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Scalable AAV Manufacturing Strategies
Integrated mRNA One-stop Solution

Dr. Xiao Pan
Sr. Director, Platform R&D Dept.

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Scalable AAV Manufacturing Strategies
SLEEK: A highly efficient transgene knock-in technology in clinically relevant cell types

Dr. John Zuris
Director of Editing Technologies – Editas Medicine

Watch Now
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