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January 6, 2012British Journal of PharmacologyOpen Access

Triptolide increases transcript and protein levels of survival motor neurons in human SMA fibroblasts and improves survival in SMA‐like mice

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Key result

Triptolide significantly increased SMN protein expression in human SMA fibroblasts and improved survival while reducing weight loss in SMA-like mice.

Why the study?

Does triptolide increase SMN protein levels and improve survival in SMA models?

Population

Motor neuronal cell line NSC34, skin fibroblasts from SMA patients, wild-type (Smn SMN2 , C57BL/6) and…

Design

Preclinical

Authors

YHYa‐Yun HsuYJYuh‐Jyh JongHTHsin‐Hung Tsai

Discussion

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Overview

Should not change SMA practice; leaves open translation of triptolide to human trials.

Structured PICO

Does triptolide increase SMN protein levels and improve survival in SMA models?

P
Population
In vitro models using NSC34 cells and human SMA fibroblasts, and in vivo models using wild-type and SMA-like mice.
I
Intervention
Triptolide (0.01 or 0.1 mg·kg(-1) ·day(-1) , i.p. in mice; pM concentrations in cells)
O
Outcome
SMN protein expression, survival rate, and weight losssurrogate

Triptolide enhances SMN protein production and increases survival in SMA mice, suggesting it may be a potential candidate for SMA therapy.

Cite This Study

Hsu et al. (2012) studied Spinal muscular atrophy (SMA). Triptolide was evaluated on SMN protein expression, survival rate, and weight loss. Triptolide significantly increased SMN protein expression in human SMA fibroblasts and improved survival while reducing weight loss in SMA-like mice.

synapsesocial.com/papers/6a962dd573e5eba5f7baba67https://doi.org/10.1111/j.1476-5381.2012.01829.x
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Also Consider

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