Knock In Mice, Genome Editing

  • Stem Cell Services

    • iPSC Generation Services

      Our iPSC reprogramming service offers a method of generating patient-specific stem cells of any lineage without using embryonic materials. We utilize various strategies to improve reprogramming technologies, including chemical and transgene reprogramming. Our protocols have been streamlined for efficient iPSC generation using viral vectors, DNA (plasmid) and mRNAs. We offer comprehensive reports suitable for publications with each of our services.

      We offer reprogramming using various vectors, including retrovirus, lentivirus, episomal plasmid, and direct delivery of synthetic mRNAs. W When deciding on a reprogramming method, we consider the cell being reprogrammed and the ability of the reprogramming method to adequately reprogram this cell type. We also assess whether the presence of integrated sequences in the iPSCs will hinder downstream application.

      Let the iPSC experts generate high-quality iPSCs and derived physiologically relevant cell line models from your healthy/disease samples. With our optimized reprogramming protocols and comprehensive characterization services, we deliver iPSCs ideal for your basic research, drug discovery, drug screening, and preclinical cell regeneration projects:

      • Highly optimized protocols with high reprogramming efficiency (>95% success rate)
      • From healthy/diseased human or non-human samples
      • iPSC generation from various donor cell types
        • human: PBMCs, fibroblast, HSC, MSCs, CD34+ cord blood, urine, and more
        • non-human: PBMC and fibroblast 
      • Integration-free (episomal/mRNA/viral-based) or retroviral reprogramming
      • Feeder-free protocols; optional feeder-dependent protocols available
      • iPSCs characterized for morphology and pluripotency markers. Additional characterization such as G-banding, RT-PCR, STR profiling, and directed differentiation is also available.
      • Fast Turnaround: 2-3 months
      • GMP iPSC Generation Available >> Learn More

      Not only can ASC further characterize your iPSCs, but our experts can also genetically engineer your iPSCs using CRISPR/Cas9 or TARGATT and differentiate the iPSCs to the cell type of your choice, including NK cells, T cells, astrocytes, cardiomyocytes, and more.

      iPSC Generation Service Categories

      iPSC Generation from Patient Samples

      iPSC Generation from Patient Samples

      Efficient iPSC generation from healthy/disease patient samples for cell line modeling, drug discovery, and drug/neurotoxicity screening. 

      iPSC Generation from Patient Samples

      GMP Grade iPSC Services & Products

      GMP Grade iPSC Services & Products

      New GMP-grade iPSC services and products are now available. Contact us today to schedule a free consultation. 

      GMP Grade iPSC Services & Products

      Allogenic and Immunocompatible iPSC Generation

      Allogenic & Immunocompatible iPSC Generation

      Full service for HLA-matched allogeneic iPSC generation and CRISPR gene editing to engineer HLA-knockouts for developing non-immunogenic iPSCs.

      Allogenic & Immunocompatible iPSC Generation

      iPSC Generation Form Non-Human Species

      iPSC Generation From Non-Human Species

      Integration-free or retroviral animal iPSC reprogramming.

      iPSC Generation From Non-Human Species

      GMP iPSC Generation and Additional Services Available >> Learn More

    • High-throughput CRISPR iPSC Genome Editing Service

      Applied StemCell (ASC) has provided stem cell and genome editing services for over 13 years, and we have worked with researchers all across the globe to engineer over 500 unique cell line models. As one of the earliest providers of CRISPR/Cas9 genome editing services, ASC has the experience and optimized protocols for Rapid Automated Cell Line Editing (RACE™) in induced pluripotent stem cells (iPSCs)!

      With our well-established high-throughput protocols, ASC's experts can produce any complex or mainstream genetic modification in your healthy or diseased iPSCs for your basic research, disease modeling, tissue engineering, regenerative medicine, or cell-based therapy research. Leverage our efficient CRISPR genome editing method to obtain your CRISPR-engineered iPS cells in just a few weeks.


      • High success rate: >98% projects completed to customer’s specifications

      • ASC can genetically modify your healthy or diseased iPSCs; control lines are available

      • Single cell cloning (clonal isolation) 

      • Homozygous or Heterozygous 

      • Automated processes for consistency and high throughput scalability

      • Optimized CRISPR iPSC protocols
      • Pluripotency maintained throughout genome editing process using high-end cell culture reagents and protocols

      • Fast turnaround time: 6-8 weeks when you select one of the ASC control lines, 3-4 months when you send in your iPSCs
      • GMP iPSC Gene Editing Available >>  Learn More

      ASC is a one-stop-shop for all your iPSC service needs. We are one of the few providers of integrated upstream iPSC generation & downstream differentiation and assay development servicesIf you are looking to engineer iPSCs in a GMP setting, we invite you to read more about our new GMP-grade iPSC service offerings

    • CRISPR/Cas9 Genome Editing

      Save Your Time, Money & Effort! Leverage our extensive expertise in CRISPR/Cas9 genome editing technologies to generate genetically modified mouse, rat, cancer and stem cell lines, with a variety of modifications in a targeted gene of interest. As one of the earliest licensees of CRISPR/Cas9 technology, we have genetically engineered > 1800 unique cell line and animal models for disease modeling, functional genomics, target identification, antibody validation, and validation for drug discovery and screening, and more. We offer affordable, comprehensive custom service with a fast turnaround time to meet the exact requirement of your projects. You can also combine it with our downstream custom assay services for a seamless project workflow.

      CRISPR/Cas9 Genome Editing Categories

      Cell Line Models

      Custom stable cell line model generation using CRISPR/Cas9 for disease modeling, antibody validation, and drug screening.

      Cell Line Models

      Mouse Models

      Custom CRISPR/Cas9 mouse engineering with a variety of genetic modifications: knockout, conditional knockout, point mutation & reporter/gene knock-in.

      Mouse Models

      Rat Models

      Custom CRISPR/Cas9 rat engineering with a variety of genetic modifications: knockout, conditional knockout, point mutation & reporter/gene knock-in.

      Rat Models

      GMP CRISPR iPSC Services >> Learn More

    • iPSC Differentiation Service

      iPSC differentiated cell lines offer the convenience of cell line models with the biorelevance of primary cells but without the sourcing difficulties and lot-to-lot variability issues associated with primary human cells. Applied StemCell (ASC) offers comprehensive service for iPSC differentiation to lineage-committed cell types thereby expanding the scope of your research, drug discovery, or drug screening projects.

      • Lineage-committed cell lines as determined by lineage-specific markers
      • Differentiation from healthy/disease iPSC lines: customer cell lines, ASC’s control iPSC lines, or genetically engineered iPSCs
      • High purity cell lines
      • Non-integrating differentiation protocols
      • Development of co-culture models with multiple lineages is available
      • Fast turnaround time
      • GMP iPSC Differentiation Services Available >> Learn More 

      Our experts can differentiate your iPSCs or you can select from our inventory of well-characterized iPSC control lines. ASC even offers upstream iPSC generation from human or non-human samples and high-throughput CRISPR genome editing services. To learn more about our affordable, custom iPSC services contact us today.

      iPSC Differentiation Service Categories

      Natural Killer Cells



      Sensory Neurons


      GABAergic Neurons

      Skeletal Muscle

      Intestinal Organoid

      T Cells

      Neural Stem Cells

      Dopaminergic Neurons


      Cholinergic Neuron

      Endothelial Cells

      Retinal Pigment Epithelium

      Hematopoietic Progenitor Cells


      Motor Neurons

      Excitatory Neurons



      Photoreceptor Cells


      GMP iPSC Differentiation & Additional Services Available >> Learn More

    • TARGATT™ Genome Editing

      Applied StemCell’s (ASC) proprietary TARGATT™ technology, enables fast and site-specific, stable integration of large DNA fragments (up to 20 kb) into an intergenic, transcriptionally active safe harbor locus with very high efficiency. The preselected locus is engineered to contain an "attP" integrase recognition landing pad where single-copy gene integration occurs when used in conjunction with an “attB” containing donor plasmid and integrase expression.

      The TARGATT™ gene editing platform is versatile and can be used for the development of large fragment knock-in cell lines, bioproduction, and library construction. This technology circumvents problems associated with random integration such as position effect, and gene silencing or instability due to the integration of multiple copies of the transgene.

      Advantages of TARGATT™ Technology:

      • Site-specific knock-in at a pre-selected safe harbor locus (no random integration)
      • Single copy gene insertion
      • Large transgene knock-in (up to 20 kb)
      • No internal genes are interrupted
      • Uninformed expression
      • High integration efficiency & High-level expression of the transgene
      • Eliminates position effect

      ASC can accurately and efficiently engineer the necessary landing pad into the cell line of your choice. Ready-to-use TARGATT TM Master Cell Lines (iPSC, HEK293, and CHO) are also available for integration of your gene of interest (GOI) at a preselected locus that has been tested for uniformed, high gene expression. Contact us today to schedule your free consultation!

      TARGATT™ Platforms

      TARGATT™ Master iPSCs for Safe Harbor Knock-in

      TARGATT™ iPSCs for Safe Harbor Knock-in

      - High-efficiency, unidirectional integration
      - Site-specific, single-copy gene knock-in
      - Well-characterized Master Cell Line

      TARGATT™ iPSCs for Safe Harbor Knock-in

      TARGATT™ CHO Master Cell lines

      TARGATT™ CHO Master Cell Lines

      - Site-specific gene insertion
      - H11 & ASC2 (A2) safe harbor, transcriptionally active genomic hotspots
      -Ideal for bioproduction

      TARGATT™ CHO Master Cell Lines

      TARGATT™ HEK293 Master Cell Lines

      TARGATT™ HEK293 Master Cell Lines

      - High knock-in efficiency
      - Site-specific, single-copy gene insertion
      - Overcomes challenges posed by random integration

      TARGATT™ HEK293 Master Cell Lines

       TARGATT™ Site-Specific Knock-in Cell Line Service

      TARGATT™ Site-Specific Knock-in Cell Line Generation Service

      - You send us your favorite cell line
      - We will insert the attP docking site at a specific loci
      - Fast turnaround time: get your cell line in as little as 3 months

      TARGATT™ Site-Specific Knock-in Cell Line Generation Service

      TARGATT™ iPSC-iNK Platform

      TARGATT™ iPSC-iNK Platform

      - >40% gene integration efficiency
      - Site-specific knock-in
      - Transfection by lipofectamine, eliminating viral manufacturing

      TARGATT™ iPSC-iNK Platform

      TARGATT™ Knock-in Mouse Models

      TARGATT™ Knock-in Mouse Models

      - TARGATT™ has been used to generate animal models
      - High-efficiency integration (up to 65%)
      - Site-specific integration
      - Germline transmitted F1 mice in 5-8 months

      TARGATT™ Knock-in Mouse Models


      Technology Comparision

      Comparing TARGATT™ and existing gene editing technologies for stable knock-in cell line development:

      TARGATT™ - For Knock-in Cell Lines

      Contact Us - TARGATT™ Services

    • iPSC Characterization Services: Check Pluripotency Markers, Chromosomal Abnormality, STR, & WGS

      Stringent molecular and functional assays are necessary to evaluate pluripotency and to rule-out genetic aberrations due to reprogramming and stress from in vitro culture in iPSC lines. Loss of genetic integrity can affect desired cellular phenotype and compromise interpretation and translation of results to a clinical setting.

      ASC offers comprehensive services needed to completely characterize your human and mouse pluripotent stem cell lines (PSCs):

      • Pluripotency and lineage-specific marker immunostaining
      • Karyotyping (Chromosome counting, G-banding, array analysis)
      • qPCR, RNA-seq
      • Trilineage differentiation potential: embryoid body (EB) formation, qRT-PCR 
      • HLA typing, whole genome sequencing (WGS), single tandem repeat (STR) genotyping, copy number variations (CNV) and more
    • iPSC-based Preclinical CRO Services

      Our comprehensive start-to-finish ipsc and stem cell platform offers downstream services to advance your iPSC-based projects to the next stage. We offer preclinical CRO solutions for drug screening, protocol development for cell regeneration and adoptive transfer model, CAR-T research, universal and immunocompatible cell line, and more. We can customize every stage of your project and find the best fit to suit your research needs.

    • GMP Grade iPSC Services & Products

      Leading iPSC CDMO, Ready-to-use iPSC GMP Master Cell Bank & GMP Cell Therapy Services

      ASC received its Drug Manufacturing License from the California Food and Drug Branch in 2021. Since then, iPSC processes have been established for cell banking and iPSC reprogramming, genome editing (CRISPR and TARGATT™), and differentiation that are compliant with FDA (US) and EMA (EU) regulations. As a CRO/CDMO committed to providing high-quality services and products, we follow quality control protocols based on ICH-Q5A/D guidelines for the development of safe iPSC and iPSC-derived products in our ISO Class 5 and 7 (Class 10,000 and 100) clean room.

      In addition to our cGMP-compliant iPSC generation service, we offer well-characterized, ready-to-use GMP-grade iPSCs (Cord Blood CD34+, Male) and cGMP-Compliant TARGATTTM Master iPSCs that can be engineered to express almost any gene of interest (e.g., CAR genes). The genome-edited cells can be further differentiated into various cell types, including NK cells, T cells neurons, RPE cells, Cardiomyocytes, Neurons, and more. Our team of experts can even work with customer-provided cell lines. We currently offer:

      Current Good Manufacturing Practice (cGMP):

      Regulations ASC complies with as a cell processing GMP facility <Located in CA, USA> 

      • 21 CFR Part 210
        • Current Good Manufacturing Practice in Manufacturing, Processing, Packing or Holding of Drugs; General
      • 21 CFR Part 211
        • Current Good Manufacturing Practice for Finished Pharmaceuticals
      • 21 CFR Part 1271
        • Human Cells, Tissues, and Cellular and Tissue-Based Products
        • Current Good Tissue Practice

      1. GMP Grade iPSCs

      1. cGMP-Grade iPSC Line: The cells are well characterized and can be genetically modified and further differentiated to various cell types.
        • Source cell type: CD34+ Cord Blood, male
        • Reprogramming method: Episomal
        • Master cell bank or working cell bank available
        • Drug master file with US FDA
        • Matching Research iPSC Line is Available

      Product QC:

      • Sterility/mycoplasma
      • Identity: SNP Trace
      • Pluripotency: image, AP stain, Oct4/TRA-1-60 stain, differentiation in vitro to 3 germ layers
      • Genetic stability: karyotyping by G-banding
      • Viability
      1. cGMP-Compliant TARGATTTM Master iPSCs: Ready to insert your GOI. High-quality iPSCs with a pre-engineered landing pad for site-specific, unidirectional gene insertion at a safe harbor locus mediated by TARGATTTM
        • Engineered from ASC’s GMP grade iPSCs using TARGATTTM (No CRISPR)
          • GMP SOP
        • All reagents are CGT grade (animal, serum-free)
        • Documentation and QC/QA follow GMP SOP
        • Certificate of Analysis provided upon request
        • All testing follows GMP requirements
        • License-ready  

      2. GMP iPSC Services

      GMP iPSCs


    • GMP TARGATT™ iPSC-iNK Platform

      iPSC Derived NK, CAR-T/CAR-NK Cell Generation

      As natural killer (NK) cell-based cancer immunotherapy research continues to grow, so has the number of potential chimeric antigen receptor (CAR)-NK therapies. Researchers continue to explore the cytotoxic capabilities of engineered NK cells, but several CAR-NK cell generation problems remain. For example, the CAR-NK cell development process takes several weeks, the patient’s NK cells may be weak and in a damaged state, or cancer cells may be created with random integration. Recent advancements in induced pluripotent stem cell (iPSC) technology have opened the door for the development of CAR-iPSCs that can be further differentiated into NK cells. These cells could potentially fix the current CAR-NK problems the research community is facing.

      Applied StemCell (ASC) combined its optimized TARGATTTM gene editing and NK differentiation technologies to establish the TARGATTTM iPSC-iNK Platform. Our expert scientists can insert your specific CAR genetic material into our iPSCs at a safe harbor locus with an efficiency ~10x better than CRISPR and further differentiate your CAR-iPSCs to high-quality NK cells. Leverage our unlimited source of iPSCs and proprietary TARGATTTM technology to safely and efficiently produce the allogenic iNK cells you need to drive your research forward.

      ASC Advantages

      • >40% gene integration efficiency
      • Site-specific knock-in
      • NK cells derived from an unlimited source of iPSCs
      • iPSC-iNK cell banks with a consistent manufacturing process
      • Safe and efficient gene editing protocols
      • Transfection by lipofectamine, eliminating viral manufacturing
      • We generate truly off-the-shelf allogeneic therapeutic cells

      As a CRO/CDMO service provider that continuously works to improve and expand its technology, services, and products, we hope to have our new cGMP TARGATT™ iPSCs available for purchase early next year. For now, we offer you fully characterized cGMP grade iPSCs from CD34+ cord blood. Contact us today to learn more.

      Why Applied StemCell?

      We understand working with iPSCs remains difficult, and you may encounter several challenges. At ASC our stem cell and gene editing experts are ready to help you every step of the way. 

      1. Send in your CAR design for review. Our experts can help you enhance your design.

      2. Our team can design research and GMP projects in parallel OR generate a matching research grade cell line for preliminary testing.

      3. Downstream Assay Services Are Available: cytotoxicity assays, in vivo testing, drug banking, and more - Inquire

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