In the field of human gene science RNA is an emerging area of interest. Scientists have recently discovered the breadth of different types of RNA, which are made by DNA, the building blocks of our genome. Discoveries in RNA research have branched into the medical field where researchers believe they can be used for gene therapy. RNA play the role of messenger in the body, delivering instructions from the DNA to proteins which control almost every aspect of the body’s functions. RNA therapeutics have many advantages over traditional medicine, chief among them is that they are able to pin point a problem with the body and fix it. A common method to utilize RNA in the body is through the use of splicing which is a great technological feat.
Key Takeaways:
- SMA is a neuromuscular condition that affects specific spinal motor neurons. Overtime the baby’s spinal cord degenerates.
- Babies born with spinal muscular atrophy rarely were known to make it past a second birthday. Today, Spinraza has changed that death sentence.
- Spinraza is a drug administered through a spinal tap, which actually fixes the garbled DNA coding responsible for the disease.
“Some lived past their toddlerhood, but never grew strong enough to run around or play with other kids and eventually succumbed to the disease.”
Read more: https://nautil.us/issue/107/the-edge/the-rise-of-rna-therapeutics
References:
- Nautil (Website)
- Alnylam Pharmaceuticals (YouTube Channel)
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