Gene Therapy for Hypophosphatasia in Mice
Author Information
Author(s): Yamamoto Seiko, Orimo Hideo, Matsumoto Tae, Iijima Osamu, Narisawa Sonoko, Maeda Takahide, Millán José Luis, Shimada Takashi
Primary Institution: Nippon Medical School
Hypothesis
Can lentiviral gene therapy effectively treat severe infantile hypophosphatasia in TNALP knockout mice?
Conclusion
A single injection of lentiviral vector expressing TNALP can significantly improve survival and health in hypophosphatasia mice.
Supporting Evidence
- Alkaline phosphatase activity in treated mice increased and remained high throughout their lives.
- Treated mice showed normal physical activity and a healthy appearance.
- Epileptic seizures were completely inhibited in treated mice.
- X-ray examination showed significantly improved mineralization in treated mice.
Takeaway
Researchers found that giving a special treatment to sick mice helped them live longer and healthier lives.
Methodology
Lentiviral vector expressing TNALP was injected into neonatal Akp2−/− mice.
Potential Biases
Potential risks of insertional mutagenesis from the viral vector.
Limitations
The study primarily involved animal models, and the long-term effects in humans are still unknown.
Participant Demographics
TNALP knockout mice, specifically Akp2−/− strain.
Statistical Information
P-Value
p<0.05
Statistical Significance
p<0.05
Digital Object Identifier (DOI)
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