What is gene therapy?

When it comes to gene therapy, we must start with "Viruses." Because viruses are inherently capable of injecting and infecting host cells with their own genetic material to replicate, the origin of gene therapy is to use viruses as vectors to after the normal genes are implanted, they are put into host cells, and the virus' replication ability is used to repair and replace abnormal genes, and then treat cancers or rare genetic diseases derived from gene defects or mutations.

The development of gene therapy

Since 2018, the US FDA and the European Union have approved more products as gene therapy drugs in these years. It makes exciting progress in the treatment of rare diseases and cancer. This progress also caused the term "Gene Therapy" to once again set off a hot topic. As one of the key drug research and development projects for the treatment of severe, cancer, or rare diseases in the future, many people may think that gene therapy is an emerging treatment that has just been developed, but in fact, the research and development of gene therapy has a history of 60 years. Along the way, it has seasoned many fail that even lead to the death of the experimenter. Until 2012, the world's first gene therapy drug was approved by the European Union. It ended the dark period of gene therapy development and brought a glimmer of light. The next few years may be the golden period of gene therapy development and breakthroughs.

Gene therapy Types: In vivo vs. Ex vivo

At present, scholars divide gene therapy into two types: Ex Vivo gene therapy and In vivo gene therapy.

Further reading: The four kinds of viral vectors which most use in gene therapy

Further reading: What Bio-buffers and Bio-detergents are suitable for the purification process of viral vectors?

Further reading: How to prepare adeno-associated viruses (AAV) vectors?

Reference:

^[1] Viral vectors: from virology to transgene expression: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2629647/

^[2] The challenge of vector development in gene therapy: https://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X1999000200001

^[3] A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene Therapies: https://www.liebertpub.com/doi/full/10.1089/hgtb.2017.096#:~:text=Specifically%2C%20lentiviruses%20can%20deliver%20up,achieve%20long%2Dterm%20stable%20expression.

^[4] Viral Vectors in Gene Therapy : https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6023384/

^[5] Gene Therapy Leaves a Vicious Cycle: https://www.frontiersin.org/articles/10.3389/fonc.2019.00297/full

^[6] Lentiviral vectors in gene therapy: their current status and future potential: https://pubmed.ncbi.nlm.nih.gov/20143172/

^[7] 病毒載體之基因治療產品於化學 製造管制研發策略指導原則: https://www.cde.org.tw/Content/Files/Knowledge/dcda94ec-bf1f-4846-84c3-fddbab0a5268.pdf

^[8] 病毒載體之基因治療產品於化學 製造管制研發策略指導原則: https://www.cde.org.tw/Content/Files/Knowledge/dcda94ec-bf1f-4846-84c3-fddbab0a5268.pdf

^[9] 藉由修飾腺相關病毒 VP1-3 建立胜肽導引式基因傳遞系統 : 國立交通大學 生物科技學院生化工程研究所

^[10] 基因治療產品化學製造管制研發指引 : 黃豐淳 博士

^[11] 基因治療：黃麗華 教授; 台大醫院肝炎研究中心

^[12] 基因治療：黃麗華 教授; 台大醫院肝炎研究中心