Massive reduction of RyR1 in muscle spindles of mice carrying recessive Ryr1 mutations alters proprioception and causes scoliosis

携带隐性Ryr1突变的小鼠肌肉梭中RyR1的大量减少会改变本体感觉并导致脊柱侧弯。

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Abstract

Muscle spindles are stretch receptors lying deep within the muscle belly involved in detecting changes in muscle length and playing a fundamental role in motor control, posture and synchronized gait. They are made up of an external capsule surrounding three to five intrafusal muscle fibres and a nuclear bag complex. Dysfunction of muscle spindles leads to abnormal proprioceptor function, which has been linked to aberrant bone and cartilage development, scoliosis, kyphosis and joint contractures. RYR1, the gene encoding the calcium release channel of the sarcoplasmic reticulum, is the most common target of mutations linked to human congenital myopathies, a condition often accompanied by skeleton alterations and joint contractures. So far, the link between RYR1 mutations, altered muscle spindles and skeletal defects has not been investigated. To this end, we investigated heterozygous mice carrying recessive Ryr1 mutations isogenic to those present in a severely affected child. Here, we show that: (i) the RyR1 protein localizes to the polar regions of intrafusal fibres and exhibits a doubled row distribution pattern, typical for junctional sarcoplasmic reticulum proteins; (ii) muscle spindles of compound heterozygous mice show structural defects; and (iii) in intrafusal muscle fibres from dHT mice, RyR1-mediated Ca(2+) release is significantly impaired and RyR1 protein content is reduced by 54%. These results support the hypothesis that Ryr1 mutations not only affect the function of extrafusal muscles, but also might affect that of intrafusal muscles. The latter may be one of the underlying causes of skeletal abnormalities seen in patients affected by recessive RYR1 mutations. KEY POINTS: Dysfunction of muscle spindles leads to abnormal proprioceptor function, which can cause abnormal bone and cartilage development, scoliosis, kyphosis and joint contractures. Muscle spindles contain intrafusal muscle fibres, which are made up of nuclear bag and nuclear chain fibres; the polar regions of intrafusal muscle fibres contain contractile filaments and sarcotubular membranes. Patients with RYR1 mutations often present skeleton alterations and joint contractures from birth. Using the dHT mouse model carrying recessive Ryr1 mutations, we investigated whether muscle spindles from dHT mice show structural defects and altered RyR1 content and function. Our results support the hypothesis that RYR1 mutations also affect the function of intrafusal muscles and this may be one of the underlying causes of the skeletal abnormalities seen in patients.

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