United Kingdom-based University of Oxford researchers developed a new treatment for spinal muscular atrophy, according to Medical Xpress.
Here are six observations:
1. The new treatment, Pip6a-PMO, involves peptide Pip6a delivering the splice-switching oligonucleotide.
2. Pip6a has the ability to deliver splice-switching oligonucleotides to many tissues in the body, even into the brain and spinal cords.
3. After injecting SMA-ridden young mice with Pip6a-PMO, they became heavier and grew faster than untreated mice.
4. The treated mice lived longer than untreated mice. One dose of 10 microgrammes per gram of weight of Pip6a-PMO allowed the young mice to live for a median 167 days, compared to 12 days for untreated mice.
5. Two doses of Pip6a-PMO resulted in mice living at least 200 days with a median survival of 457 days. This reflects a survival period that is 38 times longer than untreated mice.
6. The researchers plan to conduct a two-year study involving human patients, which is set to commence in 2017.