Even though gene therapy has been there for decades, it has faced many problems that have kept it from being widely used. The fact that adding and keeping new genes in the body’s cells is hard and expensive has made it hard to use gene therapy to its full potential.
One of the major issues with gene therapy is how hard it is to get functional genes to a large number of cells, sometimes millions, in certain organs. Also, the specific cells must be found correctly to make sure the gene gets to where it needs to go.
Once the gene is there, it needs to be turned on to make the needed protein. But keeping genes turned on is challenging as cells tend to overactive or turn to abnormal genes.
Possible Gene Therapy Challenges
Targeting Certain Genes
For gene therapy to work, it’s essential to target genes with great accuracy. It is essential for both patient safety and speed of the process that genes don’t get put into the wrong cells.
If the targeting isn’t right, the beneficial gene could end up in the patient’s germline cells, which could affect future generations.
Losing Essential Or Targeted Genes
One of the major challenges is that the target cells might lose important genes. In order for gene therapy to work for a long time, the introduced gene must stay as a fixed part of the cell’s genome.
Integration in the wrong places can cause serious problems with other genes (as seen in different gene modification studies). In a case involving gamma-c gene therapy, gene fusion into a gene that controls how fast cells divide led to leukaemia in some patients.
The ability to make money is another major problem. Many genetic diseases that could be treated with gene therapy are very rare, affecting only a few people. Even though gene therapy could save these people’s lives, the high costs of making it happen keep pharmaceutical businesses from investing.
Creating, testing, and getting government permission for new therapies is another complicated process that requires tens of money. The small number of patients may not be enough to cover these costs, leaving pharmaceutical companies unsure of how much money they will make.
Some diseases, like cancer, are quite common, so there are more people who are suffering from this. However, many gene therapy methods are customised for each patient and require that the patient’s cells be changed before they are reinfused.
Even though this personalised method might work, it’s highly expensive than any mass-produced drugs. This difference in money brings in questions that how people with average income sources can possibly afford this kind of treatment.
And heavy costing is one of the reasons why many patients hesitate taking the right treatment and this only degrades their health.
Concerns about fairness and access are also some of the challenges of gene therapy. There are different things to be concerned about when making the right decisions about the development of medicines that might not make a lot of money and the possible exclusivity of expensive treatments.
Making sure that life-changing therapies are available to everyone who needs them, no matter how much money they have, is a basic ethical problem. Finding the right balance between encouraging scientific innovation, ensuring everyone has equal access.
And putting the health of patients first is a difficult task that requires careful thought from both healthcare workers and society as a whole.
Without question, biotech has made huge impacts in different fields including medicine and gene therapies. However, there has always been certain challenges that always made different biotech companies and investors think twice before making a decision. That’s not all; the overall cost of gene therapy procedures is also another major concerns why many patients hesitate taking the treatments despite suffering from serious conditions. But as tech develops, there will definitely be certain solutions that’ll cover these challenges for good!