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巴西利卡塔-阿赫塔尔综合征:揭示发育迟缓的一种极其罕见的病因。

Basilicata-Akhtar Syndrome: Unraveling an Ultrarare Cause of Developmental Delay.

作者信息

Kulkarni Vishwanath, Chalipat Shiji, Gupta Aryan, Bhosle Akanksha, Bahal Mridu

机构信息

Pediatrics, Dr. D. Y. Patil Medical College, Hospital and Research Centre, Dr. D. Y. Patil Vidyapeeth (Deemed to be University), Pune, IND.

出版信息

Cureus. 2024 Aug 16;16(8):e67041. doi: 10.7759/cureus.67041. eCollection 2024 Aug.

DOI:10.7759/cureus.67041
PMID:39286690
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11405068/
Abstract

There is still more to learn about the etiology of extremely uncommon developmental disorders. A heterozygous or hemizygous pathogenic variation in male-specific lethal 3 (MSL3) causes the uncommon X-linked condition known as Basilicata-Akhtar syndrome, which is characterized by a global developmental delay that is evident from infancy, feeding difficulties, and muscle hypotonia. Thus far, over 40 cases have been documented. Here, we report the first case of Basilicata-Akhtar syndrome in India. A 3-year-old boy presented with global development delay. Physical examination revealed dysmorphism and hypotonia. After whole exome sequencing, exon 8 of the MSL3 gene on chromosome X showed evidence of a hemizygous single base pair deletion.

摘要

关于极其罕见的发育障碍的病因,仍有更多需要了解的地方。雄性特异性致死基因3(MSL3)的杂合或半合子致病性变异会导致一种罕见的X连锁疾病,即巴西利卡塔-阿赫塔尔综合征,其特征是从婴儿期就明显出现全面发育迟缓、喂养困难和肌肉张力减退。迄今为止,已有40多例病例记录在案。在此,我们报告印度首例巴西利卡塔-阿赫塔尔综合征病例。一名3岁男孩出现全面发育迟缓。体格检查发现畸形和肌张力减退。全外显子组测序后,X染色体上MSL3基因的第8外显子显示有半合子单碱基对缺失的证据。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/f7f16cd96e03/cureus-0016-00000067041-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/7d857e7124df/cureus-0016-00000067041-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/620a9081843e/cureus-0016-00000067041-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/f7f16cd96e03/cureus-0016-00000067041-i03.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/7d857e7124df/cureus-0016-00000067041-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/620a9081843e/cureus-0016-00000067041-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2b05/11405068/f7f16cd96e03/cureus-0016-00000067041-i03.jpg

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本文引用的文献

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