The genes present in the human body are the basic foundation of our health and development. When these genes do not function properly for some reason, many serious diseases are born. For decades, scientists have been searching for solutions that can correct or replace faulty genes. The result of this effort is Gene & Cell Therapy, which has started a new era in the medical world. Its main purpose is to improve the function of diseased cells or deliver the correct gene into the body, so that permanent cure of diseases can be possible.
Basis of Gene and Cell Therapy: How does this technology work?
Gene and cell therapy is divided into two main categories—
- Germline Gene Therapy (GGT): It is applied to reproductive cells, so that changes can be possible in future generations as well. However, it is currently banned due to ethical reasons.
- Somatic Cell Gene Therapy (SCGT): It focuses on correcting faulty genes in normal cells of the body and is currently being widely used.
SCGT can be given in two ways:
- In Vivo: Directly delivering genes to the patient’s body.
- Ex Vivo: Removing cells from the patient’s body, correcting them in the laboratory and then re-inserting them into the body.
Expansion of use: From cancer to rare diseases
Today, the use of gene and cell therapy has spread to cancer, neurological disorders, heart disease, hereditary diseases, inflammatory diseases and even some infectious diseases. According to data till 2023, about 68% of gene therapy clinical trials are focused on cancer.
Vectors: carriers of gene delivery
The biggest challenge in gene therapy is to deliver the right gene to the right cells. This involves using vectors that are either viral (e.g. Adenovirus, AAV, Lentivirus) or non-viral (e.g. plasmid DNA, nanoparticles). Viral vectors are still the most successful option because they keep the gene active in cells for a long time.
Major gene therapy products approved by the FDA and EMA
- Gendicine (2003, China): First commercial gene therapy product used to treat head and neck cancer.
- Oncorine (2005, China): First oncolytic virus therapy product that destroys cancer cells and activates the immune system.
- Glybera (2012, Europe): Treatment for rare diseases such as lipoprotein lipase deficiency.
- IMLYGIC (2015, US/Europe): First oncolytic virotherapy for melanoma.
- Strimvelis (2016, Europe): Successful gene therapy treatment for ADA-SCID (bubble-boy disease).
- Latest products like Luxturna, Zolgensma, Kymriah: For diseases like vision disorders, spinal muscular atrophy and leukemia.
Revolutionary technology: CRISPR-Cas9 and the future direction
New technology like CRISPR-Cas9 has brought an unprecedented revolution in gene editing. This technology is able to cut out specific parts of DNA and replace it with the correct sequence. Casgevy (2023) is its first FDA-approved product, which treats sickle cell disease. This technology may prove useful in even more diseases in the coming years.
Challenges and ethical questions
Although gene and cell therapy has opened up unique possibilities in medical science, many challenges remain—
- Safety: Long-term effects are not yet fully known.
- Cost: Costs up to millions of dollars, making it out of reach for most patients.
- Ethical issues: Global debate, especially around germline editing.
- Regulatory hurdles: Varying approval processes in different countries.
Conclusion: A new era of medical science
Gene and cell therapy has proven that permanent solutions to many genetic and serious diseases are possible. Although challenges remain, with technology, research and ethical balance, it has the potential to revolutionize medicine. In the coming decade, it could become the main treatment for not only rare diseases but also common diseases.