Analysis of SMN protein in umbilical cord blood and postnatal peripheral blood of neonates with SMA: a rationale for prompt treatment initiation to prevent SMA development.

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Tác giả: Hiroyuki Awano, Kazuhiro Haginoya, Tamaki Kato, Emiko Kobayashi, Mari Matsuo, Noriko Otsuki, Kayoko Saito, Toshio Saito, Yasuhiro Takeshima, Mari Urano, Mamoru Yokomura

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: England : Orphanet journal of rare diseases , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 734511

BACKGROUND: Spinal muscular atrophy (SMA) is a severe genetic neuromuscular disease caused by insufficient functional survival motor neuron protein (SMN). The SMN expression level in the spinal cord is highest during the 2nd trimester of the foetal period. We previously reported the SMN spot analysis in peripheral blood using imaging flow cytometry (IFC) as a biomarker of functional SMN protein expression. In this study, we analysed neonatal cord blood, postnatal peripheral blood, and maternal peripheral blood in presymptomatic five infants whose sibling has type 1 SMA to estimate prenatal and postnatal SMN dynamics before the onset of severe SMA. RESULTS: Data from 37 untreated patients with SMA showed that SMN-spot CONCLUSIONS: This study suggests that the status of the motor neuron pool in the spinal cord can be presumed by cord blood SMN-spot
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