Overview

Gene editing is a groundbreaking technology that allows researchers to alter an organism's DNA with precision. Among the various gene-editing methods, CRISPR-Cas9 has gained the most attention due to its efficiency, simplicity, and versatility.

CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a naturally occurring defense mechanism in prokaryotes. The CRISPR system allows bacteria to "remember" viral invaders by storing snippets of their DNA. Scientists adapted this system for gene editing, using the guide RNA (gRNA) directed Cas protein to cut DNA at specific locations and thus modify the genetic information. It can be applied both inside the body (in vivo) or outside the body (ex vivo) of a patient or donor.

CRISPR has revolutionized gene editing by offering a more precise, efficient, and accessible method. GenScript is simplifying the CRISPR-based gene editing process with user-friendly design tools, helpful protocols, and expert support, making CRISPR technology more accessible for your research and therapeutic development.

Gene Editing Workflow

Target Selection & sgRNA Design 1
sgRNA Synthesis & Screening 2
Gene Editing Toolbox 3
CRISPR/Cas Delivery 4

Introduction

GenScript offers pre-built and customized pooled CRISPR guide RNA libraries that contain precisely synthesized gRNAs designed to target every gene in human and mouse genome. These libraries can be employed in high-throughput screens to discover new targets or pathways of interest.

Designing guide RNAs (gRNAs) for CRISPR gene editing is a crucial step in ensuring the precision and efficiency of the technology. GenScript offers user-friendly online design tools for guide RNA and HDR template design with the help of the most up-to-date design algorithm.

Utilize GenSmart™ Intelligent Platform to assist your plasmid and DNA library design, optimize the design of gene sequences for better expression, and more!

Introduction

Traditional CRISPR editing techniques involve transfecting expression plasmids encoding guide RNA and/or Cas9 nuclease into host cells. By combining Cas9 protein with synthesized synthetic single guide RNAs (sgRNAs) into ribonucleoprotein (RNP) prior to delivery can significantly simplify the experimental process, while improving editing efficiency, reducing off-target effects, and avoiding immune response.

GenScript offers sgRNA synthesis service with guaranteed sequence correctness. GenCRISPR synthetic sgRNAs are more efficient, less toxic and safer than plasmid or virus based editing solutions, and are proven effective for targeted gene editing in knockout and HDR mediated knock-in experiments. We also provide custom synthesis of pegRNA for prime editing and crRNA for Cpf1/Cas12a editing systems.

GenCRISPR Synthetic sgRNA

Synthetic single guide RNA (sgRNA) is sgRNA that is chemically synthesized using the latest technological advancements. Utilizing synthetic sgRNA is a simple and cost-effective way to improve your CRISPR editing, as it offers higher editing efficiency, lower cytotoxicity, and higher lot-to-lot stability compared to IVT sgRNA.

Additionally, we offer custom synthesis of pegRNA for prime editing and crRNA for Cpf1/Cas12a editing systems.

Introduction

Through our partnership with Feng Zhang's laboratory at the Broad Institute of MIT and Harvard*, GenScript offers validated GMP Grade and RUO Grade Cas9, Cas12a and Cas13a nuclease products, sgRNA synthesis and HDR donor template synthesis services, and resources to help with leveraging CRISPR gene editing for basic research and clinical manufacturing.

One powerful application of CRISPR/Cas genome editing technology involves the precise insertion of DNA sequences via the homology-directed repair (HDR) pathway, and choosing the right HDR template is crucial for the efficacy of this approach. GenScript offers high quality, sequence verified HDR templates in both linear and circle format for maximizing the editing efficiency of your CRISPR experiments.

CRISPR/Cas9 technology was used to knock-out genes and knock-in the target sequence, resulting in gene editing cell lines that can be stably passaged. GenScript provides a variety of CRISPR gene editing cell lines. Utilizing the advantages of chemically synthesized long single-stranded gRNA, our RNP delivery system achieves both high editing efficiency and low toxicity, and support multiple delivery systems of lentivirus and plasmid to provide gene-editing cell line services with highly customizable target genes, editing regions and cell types.

GenCRISPR™ Cas Proteins

GenCRISPR™ Cas9 proteins are optimized for high CRISPR editing efficiency across various gene targets and cell types.

GenScript offers a range of Cas9 proteins, including GenCRISPR™ SpCas9, GenCRISPR™ Ultra eSpCas9, and GMP-grade Cas9.


GenCRISPR™ SpCas9

Wild-type Cas9 protein engineered for optimal CRISPR editing performance

Applications: Suitable for a wide range of CRISPR gene editing experiments.


GenCRISPR™ Ultra eSpCas9

High-fidelity Cas9 variant engineered for enhanced specificity without compromising efficiency

Applications: Ideal for CRISPR experiments that need precise editing or low off-target effects.


GMP-grade GenCRISPR™ SpCas9 and Ultra eSpCas9

Wild-type and high-fidelity Cas9 produced to meet the requirements for clinical development

Applications: Ideal for CRISPR projects that aim for therapeutic applications.

CRISPR Cas Nuclease Products

Through our partnership with Feng Zhang's laboratory at the Broad Institute of MIT and Harvard*, GenScript offers validated GMP Grade and RUO Grade Cas9, Cas12a and Cas13a nuclease products, sgRNA synthesis and HDR donor template synthesis services, and resources to help with leveraging CRISPR gene editing for basic research and clinical manufacturing.

Applications Recommendations Cat. No. Product Names
Gene KO/KI with high editing efficiency SpCas9 with NLS, his-tag (wt) RC00001New! GenCRISPR™ NLS-wtSpCas9, his-tag
RC00002-GMPNew! GMP GenCRISPR™ NLS-wtSpCas9, his-tag
Gene KO/KI with low off-target effects Ultra eSpCas9 (mt) Z03622 GenCRISPR™ Ultra eSpCas9-2NLS-Research
Z03624-GMP GenCRISPR™ Ultra eSpCas9-2NLS-GMP
Gene KO/KI following with flow cytometry or other fluorescent assays SpCas9-eGFP fusion protein Z03393 GenCrispr NLS-Cas9-EGFP Nuclease
Z03467
Gene KO/KI with lower MW Cas nucleases AsCas12a Z03502 GenCRISPR™ Cas12a (Cpf1) Nuclease
LbCas12a Z03753 GenCRISPR™ LbCas12a Nuclease
ErCas12a Z03762 GenCRISPR™ ErCas12a Nuclease
SaCas9 Z03699 GenCRISPR™ SaCas9 2NLS Nuclease
RNA cleavage LwaCas13a Z03486 GenCRISPR™ Cas13a (C2c2) Nuclease
LbuCas13a Z03742 GenCRISPR™ LbuCas13a Nuclease

New CRISPR-based Gene Editing Tools

Experience revolutionary genome editing with our new Base Editors & Prime Editors

Base editors and prime editors are gaining more attention in gene editing era. However, their complex structures, including multiple protein and RNA components, make them challenging to express and purify.

At GenScript, we have successfully produced numerous base editors and prime editors both in high purity and decent yield.

Adenine base editor (ABE)

Cytosine base editor (CBE)

Prime editor (PE) 

Catalog IVT mRNA, circRNA & saRNA Products

  • Catalog CAS enzymes mRNA

  • IVT mRNA Synthesis

In vitro transcription (IVT) refers to the process by which the DNA template containing the protein sequence is transcribed into mRNA using RNA polymerase. When this mRNA is delivered into host cells, it serves as the blueprint for protein synthesis.

GenScript provides off-the-shelf IVT CRISPR Cas mRNA products in linear mRNA and circular RNA formats for various applications.

Name Cat. No Description
eSpCas9 Circular RNA (CVB3) RP-A00017 The eSpCas9 mRNA expresses enhanced-specificity SpCas9 protein. Learn More
eSpCas9 mRNA
(Cap1, m1Ψ)
RP-A00018 The eSpCas9 mRNA expresses enhanced-specificity SpCas9 protein. It has 100% substituted with N1-methyl-pseudo Uridine for enhanced expression and reduced immunogenicity.
GenLNP-A01-eSpCas9 mRNA (m1Ψ)-TRAC sgRNA RP-A00019 The ALC0315 LNP formulation loaded with eSpCas9 mRNA (m1Ψ) and TRAC sgRNA.
GenLNP-S01-eSpCas9 mRNA (m1Ψ)-TRAC sgRNA RP-A00020 The SM102 LNP formulation loaded with eSpCas9 mRNA (m1Ψ) and TRAC sgRNA.
eSpCas9 mRNA
(Cap1, 5-MOU)
RP-A00050 The eSpCas9 mRNA expresses enhanced-specificity SpCas9 protein. It has 100% substituted with 5-methoxyuridine forenhanced expression and reduced immunogenicity.
CPF1 mRNA (N1-Methylpseudouridine/m1Ψ) RP-A00004 Cpf1 is a novel class of CRISPR-Cas DNA endonuclease. The LbCpf1/Cas12a allow efficient mutagenesis in zebrafish and Xenopus (Moreno-Mateos, et al. Nat Communication, 2017; 8: 2024). LbCpf1 increases homology-directed genome editing.
GenScript PE2/PE3 mRNA (N1-Methylpseudouridine/m1Ψ) RP-A00044 Prime editing (PE) systems minimally consist of two components: a programmable DNA nickase fused to an engineered reverse transcriptase and a pegRNA. The PE2/PE3 mRNA sequence was from publication: Nelson, et al. Nat Biotechnology, 2022; 40: 402-410.

Homology-Directed Repair (HDR) Knock-in Templates

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

* We also offer <150nt DNA Oligo Chemical Synthesis Service

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: 1-10 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: 1-20 kb

µg to g scale

Research to cGMP grade

GenScript provides over 20,000 pLentiCRISPR v2 plasmids containing gRNA sequences, and all sgRNA sequences have been verified by MIT. You can find the relevant gRNA by searching for Gene Name, Gene Symbol, or Gene ID in the database. We also provide empty vector service with vector sequences that have been verified by MIT. Multiple vector types including binary vector and all-in-one are available, and resistance screening methods can be selected or modified according to your needs.

GenScript now offers lipid nanoparticle (LNP) formulation services for the delivery of mRNA and circular RNA. Our Ready-Edit CRISPR mRNA-LNP Solutions combines popular CRISPR Cas mRNAs with different LNP formulation as off-the-shelf products to offer highly efficient, precise, and safe editing experience. Utilizing state-of-the-art lipid formulations and targeting ligands, our Targeted LNP Service ensures that the payloads are delivered directly to specific cells or tissues, thereby maximizing therapeutic impact and minimizing off-target effects.

AAV (Adeno-Associated Virus) and LVV (Lentiviral Vector) are two important viral vectors used in gene editing and therapy development. GenScript provides services covering from upstream viral vector plasmid construction to downstream viral vector packaging for research, preclinical study and clinical development.

  • ReadyEdit LNP Solutions Offerings

    1. Start your pilot project with ReadyEdit LNP Solutions

    2. Build your CRISPR components including the synthesis of mRNA, sgRNA, ssDNA, and more!

    3. LNP Formulations validated and optimized for specific CRISPR applications to significantly enhance your project's success rate.

    4. Validation with robust screening platforms to validate your gene-editing LNP designs.

    Key Benefits and Advantages of Ready-Edit LNP Platform

    Variety of ionizable lipid formulation with high encapsulation efficiency

    Targeted Delivery

    LNPs can be engineered to enhance the delivery efficiency of CRISPR system payloads

    Proven in vitro delivery & expression efficiency

    Reduced Off-Target Effects

    mRNA provides transient expression of the gene-editing tool, reducing the likelihood of off-target effects

    Variety of ionizable lipid formulation with high encapsulation efficiency

    Scalability and Production

    synthesized quickly and in large quantities using in vitro transcription

    Variety of ionizable lipid formulation with high encapsulation efficiency

    Safety Profile

    mRNA does not integrate into the host genome, reducing the risk of insertional mutagenesis

Viral Packaging Workflow

Industry Insights

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

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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

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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.

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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 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

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