Gene therapy includes injecting genomic material (foreign) into the tissue of the host. This is done to affect the expression of a gene product of cells. Initially, the primary goal of gene therapy was to treat genetic diseases, but it is currently being used to treat a wide range of ailments, including acquired and hereditary problems.
In the last 30 years, advances in genome engineering technology have made gene therapy more accessible to prevent and treat incurable diseases.
Researchers are cautiously optimistic that individuals with single-gene illnesses and complicated acquired disorders will receive safe and effective treatment. Numerous contract research firms like Scorpion focus on cell and gene-based therapies.
Gene Therapy Vs. Gene Editing
Traditional gene therapy relies on viral delivery of genes that either integrate randomly into the host genome like retroviruses or remain extrachromosomal DNA copies like AAV and express a protein absent or mutated in human disorders.
In contrast to traditional gene therapy, gene editing offers more varied tools for gene therapy, such as the ability to precisely fix point mutations, insert a different, healthy gene into a safe chromosomal site, or disrupt a gene.
The current gene-editing process uses endogenous double-strand DNA breaks and repair mechanisms.
Four types of gene-editing nuclease enzymes are available these days. Their classification is done based on their architecture:
- meganucleases
- CRISPR-associated nucleases
- transcription activator-like effector nucleases
- zinc-finger nucleases
Treating Cancer Through Gene Therapy
Gene therapy is a unique treatment option for diseases that do not have a good solution. In the past two or three decades, gene therapy for cancer treatment has made significant progress; yet, only a few medications have been authorized, and others are still in testing.
For cancer treatment, gene therapy has a higher level of safety and manageable side effects than chemotherapy. The doctors will be able to choose the best patients for treatment through gene therapy by assessing the cellular immunity and tumor genomic data.
Recent advances in the development of safe and effective gene delivery vectors and a better knowledge of nuclease activity have paved the way for future genome editing as a new therapy option for incurable disorders like cancer.
Furthermore, more affordable gene vectors will be available due to improved biological research, making gene therapy more accessible to most cancer patients.
This will transform cancer treatment in the future, moving away from generalist cancer treatment tactics and toward individualized cancer treatment based on the patient’s unique genome, immunological condition, and tumor genetic profile.
Concluding Thoughts
Gene therapy is projected to be quick, effective, low-cost, and have a greater cure rate.
Since the 1980s, gene therapy has steadily gained acceptance from the government and the general public, and in recent years, it has emerged as a viable alternative to existing treatments.
As a result, gene therapy medications, together with safe vectors and improved biotechnologies, will play a bigger role in cancer prevention and management in the future.
The future of cancer treatment using gene therapy seems promising. One can expect individualized treatment based on a specific genome and genetic profile. It would result in expedited and effective treatment with less cost and a higher cure rate.
