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Gene therapy - Coggle Diagram
Gene therapy
Current Impact
Case studies:
Jack Hogan: Hogan was one of the first people to receive Luxturna in 2017. Luxturna is a type of gene therapy used to treat a rare form of genetic blindness. After receiving therapy, his vision improved significantly.
Evie Eldridge: Eldridge received Zolgensma, a gene therapy for spinal muscular atrophy in 2019 when she was 6 months old. Zolgensma significantly improved her condition
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Future impact
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Limitations:
Limited scope: Currently, gene therapy is mainly only affective at treated single-gene based diseases and disorders, it can't treat multiple-gene based ones. And diseases caused by lifestyle factors such as type 2 diabetes can't be helped by it.
Ethical issues: In particular germline gene therapy, comes with some ethnic concerns, as it affect futures generations. Questions need to be asked, it is really okay to alter the genetic make up of children, to make them stronger, smarter or alter their appearance?
Effects that do not last: In certain cases, therapy can be temporary, requiring more and more treatments. Thats because the newly introduced genes sometimes don't stay active or get killed off.
Research
**How gene therapy works
Gene therapy is a medical field which specialises in changing and fixing faulty genes, with the aim to cure genetic disorders.
The types
Somatic Gene therapy: This practices changing the genes in some body cells that aren't producing eggs or sperms. The alterations to the genes will not end up getting passed on to the next generation.
Germline Gene therapy: This involves changing the genetic code in eggs or sperm, as a result the changes would be inherited by the offspring. However its not practiced in humans due to ethical reasons.
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Why
Why are scientists researching gene therapy?: Because gene therapy has the potential to save lives from diseases that are considered difficult and mere impossible to treat. It can be involved in treating genetic disorders, cancer treatment, chronic diseases, future prevention and precision medicine.
Why is the government funding the research?:
The government funds gene therapy research for a few key factors:
- Ethical monitoring - Having the government involved enforces research to be ethical and safe, and the risks and benefits are appropriately balanced.
- Equity - The funding can ensure that hopeful treatments can still be developed even when they're not immediately profitable.
Development:
First Gene therapy trials: The first trials of gene therapy began in the 1980s-1990s. In 1990, the first ever successful gene therapy was used to treat a four year old girl with ADA-SCID, a rare genetic immune system disorder.
Setbacks: In 1999, a young man called Jesse Gelsinger died in a gene therapy attempt. However this led to increase caution and regulation. Despite the setback, gene therapy progressed and more and more diseases were identified as suitable for gene therapy. Jesse
Recent progress 2010s-now: In the past 13 years gene therapy has advanced significantly. New technology like CRISPR-Cas9 has made it easier to modify genes.
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