Department of Medical and Surgical Pediatrics, Unit of Infantile Neuropsychiatry, University Hospital of Messina, via Consolare Valeria, Messina, 98125, Italy.
J Inherit Metab Dis. 2009 Dec;32 Suppl 1(Suppl 1):S201-5. doi: 10.1007/s10545-009-1154-4. Epub 2009 May 30.
In mammals, increased GABA in the central nervous system has been associated with abnormalities of visual evoked potentials (VEPs), predominantly manifested as increased latency of the major positive component P100. Accordingly, we hypothesized that patients with a defect in GABA metabolism, succinate semialdehyde dehydrogenase (SSADH) deficiency (in whom supraphysiological levels of GABA accumulate), would manifest VEP anomalies. We evaluated VEPs on two patients with confirmed SSADH deficiency. Whereas the P100 latencies and amplitudes for binocular VEP analyses were within normal ranges for both patients, the P100 latencies were markedly delayed for left eye (OS) (and right eye (OD), patient 1) and monocular OS (patient 2): 134-147 ms; normal <118 ms. We hypothesize that elevated GABA in ocular tissue of SSADH patients leads to a use-dependent downregulation of the major GABAergic receptor in eye, GABA(C), and that the VEP recordings' abnormalities, as evidenced by P100 latency and/or amplitude measurements, may be reflective of abnormalities within visual systems. This is a preliminary finding that may suggest the utility of performing VEP analysis in a larger sample of SSADH-deficient patients, and encourage a neurophysiological assessment of GABA(C) receptor function in Aldh5a1(-/-) mice to reveal new pathophysiological mechanisms of this rare disorder of GABA degradation.
在哺乳动物中,中枢神经系统中 GABA 的增加与视觉诱发电位 (VEPs) 的异常有关,主要表现为主要正成分 P100 的潜伏期延长。因此,我们假设 GABA 代谢缺陷、琥珀酸半醛脱氢酶 (SSADH) 缺乏症(其中 GABA 积累到超生理水平)的患者会表现出 VEP 异常。我们评估了两名确诊为 SSADH 缺乏症患者的 VEP。虽然两位患者的双眼 VEP 分析 P100 潜伏期和振幅均在正常范围内,但左眼 (OS)(右眼 (OD),患者 1)和单眼 OS(患者 2)的 P100 潜伏期明显延迟:134-147 ms;正常 <118 ms。我们假设 SSADH 患者眼组织中升高的 GABA 导致主要 GABA 能受体在眼部的使用依赖性下调,GABA(C),并且 VEP 记录的异常,如 P100 潜伏期和/或振幅测量所示,可能反映了视觉系统内的异常。这是一个初步发现,可能表明在更大的 SSADH 缺乏症患者样本中进行 VEP 分析的效用,并鼓励对 Aldh5a1(-/-) 小鼠中的 GABA(C) 受体功能进行神经生理学评估,以揭示这种罕见 GABA 降解障碍的新病理生理机制。