与过氧化物酶体生物发生障碍相关的色素性视网膜营养不良症--Zellweger 综合征谱系。

IF 0.5 Q3 MEDICINE, GENERAL & INTERNAL Oxford Medical Case Reports Pub Date : 2024-06-07 eCollection Date: 2024-06-01 DOI:10.1093/omcr/omae067
Henry Zou, Liliya Sutherland, Brooke Geddie
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引用次数: 0

摘要

色素性视网膜营养不良症(PRD)是一组遗传性疾病,涉及视杆细胞、视锥细胞和视网膜色素上皮细胞(RPE)的进行性变性,可发展为色素性视网膜病变(PR)。我们介绍了一例患有色素性视网膜病变的女性儿科患者,她的 PRPH2 和 PEX1 基因存在致病变异。患者伴有黄斑水肿和继发性视力损害。治疗包括连续注射地塞米松玻璃体内植入剂和局部使用多佐胺。PEX1 基因突变与过氧化物酶体生物发生障碍-Zellweger 综合征谱系(PBD-ZSS)以及由此导致的视网膜营养不良症有关。PRPH2 基因突变可能在黄斑水肿和 PRD 中起作用,因为它与黄斑变性、脉络膜缺陷和感光器功能障碍有关。在本病例中,我们回顾了在 PRD 中起潜在病因作用的多种基因突变,并讨论了护理管理问题。
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Pigmentary retinal dystrophy associated with peroxisome biogenesis disorder-Zellweger syndrome spectrum.

Pigmentary retinal dystrophy (PRD) is a group of inherited disorders involving the progressive degeneration of rod and cone photoreceptors and the retinal pigment epithelium (RPE), which can progress to pigmentary retinopathy (PR). We present a case of PRD in a female pediatric patient who has pathogenic variants in the PRPH2 and PEX1 genes. The patient has associated macular edema and secondary visual impairment. Treatment has included serial dexamethasone intravitreal implant injections and topical dorzolamide. The PEX1 gene mutation is associated with peroxisome biogenesis disorder-Zellweger syndrome spectrum (PBD-ZSS) and resulting retinal dystrophies. The PRPH2 mutation may play a role in macular edema and PRD, as it is implicated in macular degeneration, choroid defects, and photoreceptor dysfunction. In this case, we review multiple gene mutations playing potential etiologic roles for PRD and discuss care management.

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来源期刊
Oxford Medical Case Reports
Oxford Medical Case Reports MEDICINE, GENERAL & INTERNAL-
CiteScore
0.90
自引率
0.00%
发文量
125
审稿时长
19 weeks
期刊介绍: Oxford Medical Case Reports (OMCR) is an open access, peer-reviewed online journal publishing original and educationally valuable case reports that expand the field of medicine. The journal covers all medical specialities including cardiology, rheumatology, nephrology, oncology, neurology, and reproduction, comprising a comprehensive resource for physicians in all fields and at all stages of training. Oxford Medical Case Reports deposits all articles in PubMed Central (PMC). Physicians and researchers can find your work through PubMed , helping you reach the widest possible audience. The journal is also indexed in the Web of Science Core Collection . Oxford Medical Case Reports publishes case reports under the following categories: Allergy Audiovestibular medicine Cardiology and cardiovascular systems Critical care medicine Dermatology Emergency medicine Endocrinology and metabolism Gastroenterology and hepatology Geriatrics and gerontology Haematology Immunology Infectious diseases and tropical medicine Medical disorders in pregnancy Medical ophthalmology Nephrology Neurology Oncology Paediatrics Pain Palliative medicine Pharmacology and pharmacy Psychiatry Radiology, nuclear medicine, and medical imaging Respiratory disorders Rheumatology Sexual and reproductive health Sports Medicine Substance abuse.
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