探索巨囊-微结肠-肠道蠕动减弱综合征的复杂性:基因研究的启示。

IF 0.8 Q4 GASTROENTEROLOGY & HEPATOLOGY
Clinical Journal of Gastroenterology Pub Date : 2024-06-01 Epub Date: 2024-03-09 DOI:10.1007/s12328-024-01934-x
Prasad K V Devavarapu, Kalyan Ram Uppaluri, Vrushabh Anil Nikhade, Kalyani Palasamudram, Kavutharapu Sri Manjari
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引用次数: 0

摘要

巨结肠小肠蠕动减弱综合征(MMIHS)是一种不常见的常染色体隐性遗传疾病,会影响膀胱和肠道的肌肉。与 MMIHS 基因突变相关的最常见基因是 ACTG2、LMOD1、MYH11、MYL9、MYLK 和 PDCL3。然而,MMIHS 的完整基因图谱仍有待全面了解。MMIHS 的诊断具有挑战性。然而,产前诊断技术(如超声波和胎儿尿液分析)的进步提高了早期发现该综合征的能力。有针对性的下一代测序(NGS)和其他诊断测试也能诊断 MMIHS。MMIHS 的治疗包括处理严重的肠道运动障碍,这通常需要全肠外营养(TPN),而全肠外营养可能导致肝毒性和营养缺乏等并发症。多肠道移植和肠道移植已成为治疗选择,为改善治疗效果和肠内自主提供了可能。了解 MMIHS 的遗传基础对于个性化治疗至关重要。虽然预后各不相同,但及时干预和仔细监测可提高患者的预后。遗传学研究为我们了解 MMIHS 的分子机制提供了宝贵的资料。这些研究发现了平滑肌细胞发育和功能相关基因的突变。研究还表明,MMIHS 与控制肌肉收缩的信号通路缺陷有关。对 MMIHS 遗传学的持续研究有望揭开 MMIHS 的复杂面纱,改善患者的生活。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Exploring the complexities of megacystis-microcolon-intestinal hypoperistalsis syndrome: insights from genetic studies.

Megacystis microcolon intestinal hypoperistalsis syndrome (MMIHS) is an uncommon genetic disorder inherited in an autosomal recessive pattern that affects the muscles that line the bladder and intestines. The most common genes associated with MMIHS mutations are ACTG2, LMOD1, MYH11, MYL9, MYLK, and PDCL3. However, the complete genetic landscape of MMIHS still needs to be fully understood. The diagnosis of MMIHS can be challenging. However, advances in prenatal and diagnostic techniques, such as ultrasound and fetal urine analysis, have improved the ability to detect the syndrome early. Targeted next-generation sequencing (NGS) and other diagnostic tests can also diagnose MMIHS. The management of MMIHS involves addressing severe intestinal dysmotility, which often necessitates total parenteral nutrition (TPN), which can lead to complications such as hepatotoxicity and nutritional deficiencies. Multivisceral and intestinal transplantation has emerged as therapeutic options, offering the potential for improved outcomes and enteral autonomy. Understanding the genetic underpinnings of MMIHS is crucial for personalized care. While the prognosis varies, timely interventions and careful monitoring enhance patient outcomes. Genetic studies have given us valuable insights into the molecular mechanisms of MMIHS. These studies have identified mutations in genes involved in the development and function of smooth muscle cells. They have also shown that MMIHS is associated with defects in the signaling pathways that control muscle contraction. Continued research in the genetics of MMIHS holds promise for unraveling the complexities of MMIHS and improving the lives of affected individuals.

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来源期刊
Clinical Journal of Gastroenterology
Clinical Journal of Gastroenterology GASTROENTEROLOGY & HEPATOLOGY-
CiteScore
2.00
自引率
0.00%
发文量
182
期刊介绍: The journal publishes Case Reports and Clinical Reviews on all aspects of the digestive tract, liver, biliary tract, and pancreas. Critical Case Reports that show originality or have educational implications for diagnosis and treatment are especially encouraged for submission. Personal reviews of clinical gastroenterology are also welcomed. The journal aims for quick publication of such critical Case Reports and Clinical Reviews.
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