Gene Therapy Cargo Development Service
Gene therapy cargo include a series of DNA, RNA, proteins and other therapeutic agents that are delivered to target cells to exert therapeutic effects in vivo. Our company provides high-end research services and integral platforms to help you be at the forefront of the development of gene therapy cargoes for Autoimmune Diseases & Inflammation.
Gene Therapy Cargoes in Autoimmune Diseases & Inflammation
A large number of therapeutic targets have been identified in the long-term study of the mechanism of autoimmune diseases. The development of agents in the form of gene therapy for these targets has also gained inspiration from other disciplines, including DNA, RNA, RNA-protein complexes, etc. These diverse forms provide many options for the therapeutics of autoimmune diseases with different mechanisms.
Fig.1 Schematic representation of gene therapies involved in the main central nervous system diseases (Luo M., et al., 2022)
DNA Therapies
Synthetic gene fragments and plasmids are the primary DNA cargoes, carrying artificially designed gene segments. DNA fragment synthesis is a simple and fast method, but in comparison, DNA fragments have limitations in terms of length, whereas plasmids can maintain transfection efficiency without significant impact even when reaching a size of 20kb. Additionally, the probability of plasmid DNA causing immunogenic safety issues is lower.
RNA Therapies
Various RNA cargoes are employed in gene therapy with the disability of disturbing the genome. When it comes to interfering with the expression of a particular disease-causing gene and inducing changes in its protein product, microRNAs, siRNA, shRNA and PIWI-interaction RNA (piRNA) can all achieve this goal. While mRNA therapy is another way to convert protein products with higher transfection efficiency and lower risk of genome invasion compared to DNA plasmids.
Antisense Oligonucleotide Therapies
Antisense Oligonucleotides (ASOs) are synthetic DNA or RNA molecules designed to complementarily bind to the RNA of a pathogenic gene, thereby inhibiting its expression levels. Through various modifications, ASOs exhibit high cellular permeability and can disrupt RNA transcription and/or RNA-protein interactions in a transcript's degradation-dependent or independent manner.
CRISPR Gene Editing Therapies
The CRISPR-Cas system holds promising prospects in gene therapy. The complex of Cas proteins and guide RNA insert or delete target genes. The iteratively engineered dead Cas protein (dCas) has emerged as a more intriguing option, as it is unable to cleave the genome, avoiding alterations to the genetic material. Instead, the additional functional domains modulate gene expression through changes in epigenetic modifications.
Our Services
The diversity of gene therapy cargoes makes it exceedingly possible to intervene in Autoimmune Diseases & Inflammation through gene therapy. Our company provides latest technologies and methodologies for the gene therapy cargo development services with efficiency and reliability, including but not limited as following:
DNA Fragment Synthesis
- Target Sequence Design
- Plasmid Construction
- Expression and Purification
- Sequence Analysis
- Delivery Vector Construction
RNA Fragment Synthesis
- Target Sequence Design
- Plasmid Construction
- RNA Transcription
- RNA Purification
- Delivery System Design
ASO Synthesis
- Target Determination
- ASO Sequence Design
- ASO Sequence Modification
- Delivery System Design
- Bioactivity Evaluation
CRISPR-Cas System Development
- CRISPR Gene Editing Design
- Clonal Screening and Expansion
- CRISPR Gene Sequencing
- Gene Editing Efficiency Measurement
Workflow for Gene Therapy Cargo Development
Target Determination
Fragment Amplification or Synthesis
Delivery System Selection & Optimization
In Vitro & In Vivo Assessment
Our Advantages
- Professional team with customizable studies
- Complete set of high-tech equipment
- Mature development and application procedure
- Laboratories equipped with state-of-the-art instruments
Composed of genetics experts and genetic engineering researchers with extensive research and laboratory resources worldwide, our company is committed to providing you with customized solutions to meet the unique requirements of different projects for Autoimmune Diseases & Inflammation. If you are interested in our services, please don't hesitate to contact us.
Reference
- Luo, Meihua et al. "Delivering the Promise of Gene Therapy with Nanomedicines in Treating Central Nervous System Diseases." Advanced science (Weinheim, Baden-Wurttemberg, Germany) 26 (2022): e2201740.