Upcoming developments in gene therapy

Gene therapy is a relatively new medical treatment which is designed to alleviate diseases by modifying the genes, or sometimes altering the production of proteins by the faulty genes. This method aims to treat conditions that have long been deemed untreatable through traditional methods. Scientists first demonstrated the feasibility of incorporating new genetic functions into mammalian cells in the late 1960s. 

1. Early Years (1900s-1980s)

This period of time brought imperative discoveries in genetics, where DNA was identified and other breakthroughs such as gene manipulation techniques were discovered during these years. However, it was only in the late 1980s where the potential that gene therapy has in the lives of others was fully understood. Researchers began to brainstorm ideas on how genetic defects could be corrected by introducing functional genes, which at the time was a revolutionary idea. 

2. First Human Trial (1990) 

The very first attempt at gene therapy for metabolic disease began in 1970. However, this attempt failed to produce the changes that the scientists desired. Using this attempt as a means of gaining valuable knowledge, the science behind gene therapy advanced rapidly. It helped Ashanti DeSilva, a twenty-six month old who was diagnosed with SCID after being affected by a ceaseless series of infections. Scientists used a technique of isolating and transferring T cells using a retroviral vector and this procedure was finally accepted. Four months later, Ashanti DeSilva became the first to receive the treatment. Four months later, a nine year old called Cynthia Cutshall followed with the treatment. This trial was seen as a success since there were no serious side effects and both children grew up normally into adulthood. 

3. Improvements in Viral Vectors 

There were many challenges and horror stories in the development of gene therapy, such as an 18 year old patient tragically dying in 1999 due to complications in a gene therapy trial. Despite this, scientists pursued and in 2010, there were advancements in viral vectors. Viral vectors are engineered viruses which are used to deliver genes into human cells. The development in these vectors allowed the creation of new designs which are safer and more efficient. This successfully led to treatments for numerous genetic disorders. 

4. CRISPR and Genetic Editing 

CRISPR is a technology that is used to edit genes. It is often described as using ‘a pair of molecular scissors’ and is considered to be the most precise, cost effective and quickest way to edit genes. It allows scientists to ‘cut and paste’ sections of DNA. This advancement could have the potential to cure genetic disorders such as sickle cell disease and perhaps even certain cancers. 

5. The Future of Gene Therapy 

After the introduction of CRISPR gene therapy and genetic editing, they have been linked to numerous ground-breaking advancements in healthcare. 

• Highly personalised treatment plans will become more common. Advancements in sequences, CRISPR and bioinformatics have deepened the understanding of individual genomes. There are hopes of treatments, which are not only disease-specific but patient-specific. By the end of 2024, there may be more AI-driven tools to create highly sophisticated, specific genetic therapy treatment plans. 

• Optimizing Drug Delivery will hopefully improve efficiency. With the newly developed delivery systems, such as targeted nanoparticles and viral vectors, gene therapy can be administered more efficiently to specific cells or tissues. 

• There are numerous advancements surrounding the rapid growth of AI. Numerous tools and clinical trial designs have been cantered around the development of AI. 

Conclusion 

Gene therapy, first proposed in the late 1960s, is a rapidly evolving field, holding the future of technological advancements to better healthcare for patients. Not only does it cure diseases which were once deemed incurable, but it improves the efficiency of numerous tools. The future of gene therapy has the potential to treat certain cancers and improve treatment plans which can ideally improve the lives of patients.

Written by Achinthya

Moderated by Adelene

References

1. Moawad, H. (2022). An Overview of Gene Therapy. [online] Verywell Health. Available at: https://www.verywellhealth.com/gene-therapy-5214362.

2. Biomol GmbH - Life Science Shop. (n.d.). The First Human Gene Therapy. [online] Available at: https://www.biomol.com/resources/biomol-blog/the-first-human-gene-therapy.

3. Mohsen Danaeifar (2022). Recent advances in gene therapy: genetic bullets to the root of the problem. doi:https://doi.org/10.1007/s10238-022-00925-x.

4. Pal, M. (2018). 7 Gene Therapy Examples & Applications in Healthcare. [online] The Kolabtree Blog. Available at: https://www.kolabtree.com/blog/7-gene-therapy-examples-applications-in-healthcare#:~:text=7%20Gene%20Therapy%20Examples%20%26%20Applications%20in%20Healthcare.

5. CSL. (n.d.). A Gene Therapy Timeline. [online] Available at: https://www.csl.com/we-are-csl/vita-original-stories/2022/a-gene-therapy-timeline.