Adeno-Associated Virus Solutions
Luminescent, genomic and proteomic solutions for AAV analytical method development.
Novel solutions to support lentiviral vector-based gene therapy development.
Enzymes, kits and genomics tools to support RNA biology, mRNA therapeutic development and more!
Introduction to Gene Therapy
Mutations and deletions in the genome can cause many serious disorders and diseases. The goal of gene therapy research is to create a treatment that corrects these genomic errors through the insertion of genetic material that corrects or replaces the mutated or missing gene, or by modulating the target gene expression. There are several different delivery mechanisms that are used in gene therapy research including viral vectors and lipid nanoparticles.
Viral vectors are attractive delivery mechanisms for gene therapy because their natural infectivity gives them high cellular uptake. Two of the most common viral vectors are adeno-associated virus (AAV) and lentivirus (LV). These vectors have unique characteristics that can be exploited for different applications.
AAV vectors are non-integrating viruses, which means they do not integrate with the genome of the host cell and have episomal expression that results in transient therapeutic gene expression. In contrast, LV vectors are integrating viruses and result in permanent, stable therapeutic gene expression. LV vectors can accommodate relatively large target genes, whereas AAV vectors are limited to genes 4kb or smaller.
RNA-based technologies can be used in gene therapy to target DNA, RNA and protein, as well as to express proteins. For targeting nucleic acids, RNA therapies can use single-stranded, anti-sense oligonucleotides (ASOs), or target the RNA interference (RNAi) pathway using small double-stranded molecules such as small interfering RNAs (siRNAs) or micro RNAs (miRNAs).
Both ASOs and RNAi are used to interrupt the translation process, either by preventing mRNA translation (ASOs) or by degrading the mRNA (RNAi). RNA therapies can also be used to target proteins using an RNA aptamer, which binds to a specific site on the protein and modulates its functions. Finally, mRNA therapies are being explored for vaccine use against both cancer and infectious diseases.