拯救卡纳万病：在正确的时间改造错误的细胞。 - Suppr

Rescuing Canavan disease: engineering the wrong cell at the right time.

作者信息

Baslow Morris H

机构信息

Center for Biomedical Imaging and Neuromodulation, Nathan Kline Institute for Psychiatric Research, 140 Old Orangeburg Road, Orangeburg, NY, 10962, USA.

出版信息

J Inherit Metab Dis. 2017 Sep;40(5):627-628. doi: 10.1007/s10545-017-0038-2. Epub 2017 Apr 3.

DOI:10.1007/s10545-017-0038-2

PMID:28374337

Abstract

摘要

相似文献

Rescuing Canavan disease: engineering the wrong cell at the right time.拯救卡纳万病：在正确的时间改造错误的细胞。

J Inherit Metab Dis. 2017 Sep;40(5):627-628. doi: 10.1007/s10545-017-0038-2. Epub 2017 Apr 3.

N-Acetylaspartate Synthase Deficiency Corrects the Myelin Phenotype in a Canavan Disease Mouse Model But Does Not Affect Survival Time.N-乙酰天门冬氨酸合成酶缺乏症纠正了卡纳万病小鼠模型中的髓鞘表型，但不影响存活时间。

J Neurosci. 2015 Oct 28;35(43):14501-16. doi: 10.1523/JNEUROSCI.1056-15.2015.

N-acetylaspartate supports the energetic demands of developmental myelination via oligodendroglial aspartoacylase.N-乙酰天门冬氨酸通过少突胶质细胞天冬氨酸酰基转移酶支持发育性髓鞘形成的能量需求。

Neurobiol Dis. 2016 Dec;96:323-334. doi: 10.1016/j.nbd.2016.10.001. Epub 2016 Oct 4.

Aspartoacylase is restricted primarily to myelin synthesizing cells in the CNS: therapeutic implications for Canavan disease.天冬氨酸酰基转移酶主要局限于中枢神经系统中合成髓磷脂的细胞：对卡纳万病的治疗意义。

Brain Res Mol Brain Res. 2002 Nov 15;107(2):176-82. doi: 10.1016/s0169-328x(02)00490-4.

Restoration of aspartoacylase activity in CNS neurons does not ameliorate motor deficits and demyelination in a model of Canavan disease.在Canavan病模型中，恢复中枢神经系统神经元中天冬氨酸酰基转移酶活性并不能改善运动功能障碍和脱髓鞘。

Mol Ther. 2005 May;11(5):745-53. doi: 10.1016/j.ymthe.2005.01.006.

Myelin lesion in the aspartoacylase (Aspa) knockout rat, an animal model for Canavan disease.天冬氨酸酶（Aspa）敲除大鼠的髓鞘病变，一种 Canavan 病的动物模型。

Exp Anim. 2024 Jul 9;73(3):347-356. doi: 10.1538/expanim.23-0089. Epub 2024 Mar 28.

Defective myelin lipid synthesis as a pathogenic mechanism of Canavan disease.髓鞘脂质合成缺陷作为卡纳万病的致病机制

Adv Exp Med Biol. 2006;576:145-63; discussion 361-3. doi: 10.1007/0-387-30172-0_10.

A novel aspartoacylase (ASPA) gene mutation in Canavan disease.一种新发现的与Canavan病相关的天冬氨酸酰基转移酶（ASPA）基因突变。

Fetal Pediatr Pathol. 2012 Aug;31(4):236-9. doi: 10.3109/15513815.2011.650292. Epub 2012 Apr 2.

Docking, molecular dynamics and free energy studies on aspartoacylase mutations involved in Canavan disease.关于参与卡纳万病的天冬氨酸酰基转移酶突变的对接、分子动力学和自由能研究。

J Mol Graph Model. 2017 Jun;74:44-53. doi: 10.1016/j.jmgm.2017.03.011. Epub 2017 Mar 19.

Effects of AAV-2-mediated aspartoacylase gene transfer in the tremor rat model of Canavan disease.腺相关病毒2型介导的天冬氨酸酰基转移酶基因转移在卡纳万病震颤大鼠模型中的作用。

Brain Res Mol Brain Res. 2005 Apr 27;135(1-2):112-21. doi: 10.1016/j.molbrainres.2004.12.007.

引用本文的文献

The full spectrum of MRI findings in 18 patients with Canavan disease: new insights into the areas of selective susceptibility.18 例脑腱黄瘤病患者的 MRI 全谱表现：选择性易感性领域的新见解。

Neuroradiology. 2024 Oct;66(10):1829-1835. doi: 10.1007/s00234-024-03388-x. Epub 2024 Jun 17.

The pathogenesis of, and pharmacological treatment for, Canavan disease.脑腱黄瘤病的发病机制与药物治疗。

Drug Discov Today. 2022 Sep;27(9):2467-2483. doi: 10.1016/j.drudis.2022.05.019. Epub 2022 May 27.

Cell-Based Therapy for Canavan Disease Using Human iPSC-Derived NPCs and OPCs.使用人诱导多能干细胞衍生的神经前体细胞和少突胶质前体细胞治疗Canavan病的细胞疗法。

Adv Sci (Weinh). 2020 Oct 29;7(23):2002155. doi: 10.1002/advs.202002155. eCollection 2020 Dec.

Increasing N-acetylaspartate in the Brain during Postnatal Myelination Does Not Cause the CNS Pathologies of Canavan Disease.产后髓鞘形成期间脑内N-乙酰天门冬氨酸增加不会引发卡纳万病的中枢神经系统病变。

Front Mol Neurosci. 2017 Jun 2;10:161. doi: 10.3389/fnmol.2017.00161. eCollection 2017.

本文引用的文献

Redirecting -acetylaspartate metabolism in the central nervous system normalizes myelination and rescues Canavan disease.中枢神经系统中 -乙酰天门冬氨酸代谢的重定向可使髓鞘形成正常化并挽救 Canavan 病。

JCI Insight. 2017 Feb 9;2(3):e90807. doi: 10.1172/jci.insight.90807.

Molecular identification of aspartate N-acetyltransferase and its mutation in hypoacetylaspartia.天门冬氨酸 N-乙酰转移酶的分子鉴定及其在低乙酰天冬氨酸血症中的突变。

Biochem J. 2009 Dec 14;425(1):127-36. doi: 10.1042/BJ20091024.

Are astrocytes the missing link between lack of brain aspartoacylase activity and the spongiform leukodystrophy in Canavan disease?星形胶质细胞是天冬氨酸酰基转移酶活性缺乏与卡纳万病海绵状脑白质营养不良之间缺失的环节吗？

Neurochem Res. 2009 Sep;34(9):1523-34. doi: 10.1007/s11064-009-9958-z. Epub 2009 Mar 25.

Absence of N-acetylaspartate in the human brain: impact on neurospectroscopy?人脑中N-乙酰天门冬氨酸的缺失：对神经光谱学有何影响？

Ann Neurol. 2001 Apr;49(4):518-21.

Functions of N-acetyl-L-aspartate and N-acetyl-L-aspartylglutamate in the vertebrate brain: role in glial cell-specific signaling.N-乙酰-L-天冬氨酸和N-乙酰-L-天冬氨酰谷氨酸在脊椎动物大脑中的功能：在神经胶质细胞特异性信号传导中的作用。

J Neurochem. 2000 Aug;75(2):453-9. doi: 10.1046/j.1471-4159.2000.0750453.x.

Expression of aspartoacylase activity in cultured rat macroglial cells is limited to oligodendrocytes.培养的大鼠大胶质细胞中天冬氨酸酰基转移酶活性的表达仅限于少突胶质细胞。

J Mol Neurosci. 1999 Aug-Oct;13(1-2):47-53. doi: 10.1385/JMN:13:1-2:47.

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Rescuing Canavan disease: engineering the wrong cell at the right time.

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