Gene Therapy

What is Gene Therapy?

Depending on the treatment, the target of gene therapy may differ. On one hand, germ or stem cells as a target introduce heritable changes to the genome, which could find their use in hereditary disease. Germline gene therapy is subject to many ethical debates due to unforeseeable risks and technical difficulties. On the other hand, somatic cells are often used for genetic disorders such as immunodeficiencies, hematological disorders such as hemophilia, and thalassemia, or cystic fibrosis, with many clinical trials currently underway. 

Modes of Gene Transfer 

Gene therapy distinguishes between several modes of transfer: 
First, gene delivery may occur by inserting DNA in a naked or complex-loaded form into the host cell. Though the transfer of naked DNA, e.g. by complexing it with Calcium ions or complexes is probably one of the oldest technique to transfect it is hampered by some limitations such as low in vivo availability. However, with the advent of the genome editing tool CRISPR-Cas9 a novel approach with great potential was established. It makes use of the bacterial (S. pyogenes) nuclease Cas9 which is associated with its anti-viral defense system, and load it with target DNA and a synthetic guide-RNA to modify the host genome at any desired location. However, since Cas9 is of bacterial e.g. non-human origin it has the potential to trigger an immune response. To monitor immune responses against this endonuclease we developed two PepMix peptide subpools covering Cas9 antigens. 
 Second, vector-based gene transfer, also known as viral-based gene transfer makes use of a virus to deliver and replicate DNA in the host cell. Different viruses e.g. measles, lentivirus, or modified HSV1 have been and are being used as gene delivery vehicles. 
Adenoviridae (AdV) and Adeno-associated viruses (AAV) are the most commonly researched vectors for anti-cancer therapy or for vaccine development against infectious diseases such as Ebola or SARS-CoV-2. For instance, human AdV type 5 & 26 and AdVs derived from chimpanzees are commonly used artificial vectors with its replication machinery inhibited, and thus exclusively allowing gene transfer while preventing multiplication. 
 Moreover, adeno-associated viruses (AAV) can infect both dividing and quiescent cells, and exist in an extrachromosomal, thus not integrating state, making them also state-of-the-art candidates for gene therapy. So far they are being employed in the treatment for retinal dystrophy lipoprotein lipase deficiency, hemophilia A, and spinal muscular atrophy.

Amongst monitoring efficiency of viral vectors, the detection of potential anti-vector immunity by the host is crucial for effective gene therapy development and safety. We have developed multiple peptide tools in the form of PepMix peptide pools, PepStar Microarrays, or Single Antigen Peptides, which cover the following antigens: 
  • Capsid proteins, such as penton and hexon protein for a human and chimpanzee Adv 
  • Capsid proteins for Adeno-Associated Virus 5

JPT's peptide tools to study gene therapy  

JPT's Peptide Formats

Cellular Immune Response

PepMix™ Peptide Pools  
  • Antigen specific stimulation of T-cells
  • Immune monitoring of high-risk patients
  • Qualification of immunodominant antigens
  • Validating clinical T-cell assays

PepMixes™ for the following Antigens:

Tailored PepMix™ Peptide Pools for your specific needs!

Humoral Immune Response

  • Immune Monitoring of humoral responses
  • Evaluation of co-infection
  • Detection of epitopes and epitope spreading

PepStar™ Peptide Microarrays for the following Antigens:
You define content and layout, we provide economic and fast production in our regulated clean-room environment. We also offer our assay and analysis service using your samples with your tailored peptide microarray.

Clinical Peptides

Clinical Peptides
JPT’s Clinical Peptides product lines Clinical Grade and ISO Plus are produced in production environments that are regulated by an enhanced ISO 9001:2015 quality management system for the stringent product requirements of immunotherapy as well as vaccine and drug development. Depending on the specifics of the immunotherapy concept to be applied, the resulting products have been shown to be applicable in clinical applications. mune Monitoring of humoral responses



  • LOAd703, an oncolytic virus-based immunostimulatory gene therapy, combined with chemotherapy for unresectable or metastatic pancreatic cancer (LOKON001): results from arm 1 of a non-randomised, single-centre, phase 1/2 study
    Musher et al., The Lancet Oncology (2024) 
    Products used: PepMix Human Adenovirus 26 (Hexon Protein) and Human Adenovirus 26 (Penton Protein)
  • Gene Transfer in Adeno-Associated Virus Seropositive Rhesus Macaques Following Rapamycin Treatment and Subcutaneous Delivery of AAV6, but Not Retargeted AAV6 Vectors
    Stone et al., Human Gene Therapy (2022)
  • Modular capsid decoration boosts adenovirus vaccine-induced humoral and cellular immunity against SARS-CoV-2
    Dicks et al., BioRxiv (2022)
  • Characterization of Recombinant Gorilla Adenovirus HPV Therapeutic Vaccine PRGN-2009
    Pellom, JCI Insight (2021)
  • Implementation of Adenovirus-Mediated Pulmonary Expression of Human ACE2 in HLA Transgenic Mice Enables Establishment of a COVID-19 Murine Model for Assessment of Immune Responses to SARS-CoV-2 Infection
    Chitlaru et al., Pathogens, (2021)
  • Antigenic Competition in the Generation of Multi-Virus-Specific Cell Lines for Immunotherapy of Human Cytomegalovirus, Polyomavirus BK, Epstein-Barr Virus and Adenovirus Infection in Haematopoietic Stem Cell Transplant Recipients
    Roubalová, Immunol Lett. (2020)
  • Adenovirus Vector-Based Vaccines Confer Maternal-Fetal Protection against Zika Virus Challenge in Pregnant IFN-αβR−/− Mice
    Larocca, Clinical and Translational Report (2019)
  • Replication Deficient Human Adenovirus Vector Serotype 19a/64: Immunogenicity in Mice and Female Cynomolgus Macaques
    Ragonnaud et al., Vaccine (2018) 
  • Development of a Replication-Deficient Adenoviral Vector-Based Vaccine Candidate for the Interception of HPV16- and HPV18-Induced Infections and Disease
    Khan et al., International Jounal of Cancer (2017)
  • Breadth of T Cell Responses after Immunization with Adenovirus Vectors Encoding Ancestral Antigens or Polyvalent Papillomavirus Antigens
    Ragonnaud et al., Scandinavian Journal of Immunology (2017)
  • Chimpanzee Adenovirus– and MVA-Vectored Respiratory Syncytial Virus Vaccine is Safe and Immunogenic in Adults
    Green et al., Science Translational Medicine. (2015)
Application Notes

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