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:

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

-> Peptide Microarrays 
  • 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
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References

References

Application Notes

Application Notes

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