Long-term preservation of photoreceptor function and structure following early-stage treatment by AAV-mediated gene augmentation in canine model of NPHP5 Leber congenital amaurosis

Aguirre Gustavo D.; Cideciyan Artur V.; Boye Sanford L.; Iwabe Simone; Dufour Valerie; Swider Malgorzata; Roszak Karolina; Hauswirth William W.; Jacobson Samuel G.; Beltran William A.

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Long-term preservation of photoreceptor function and structure following early-stage treatment by AAV-mediated gene augmentation in canine model of NPHP5 Leber congenital amaurosis

机译：通过AAV介导的基因增强在NPHP5 Leber先天性神经症犬模型中的早期阶段治疗后的光感受器函数和结构的长期保存

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来源
《Investigative ophthalmology & visual science 》 |2018年第9期| 共3页
作者
Aguirre Gustavo D.; Cideciyan Artur V.; Boye Sanford L.; Iwabe Simone; Dufour Valerie; Swider Malgorzata; Roszak Karolina; Hauswirth William W.; Jacobson Samuel G.; Beltran William A.;
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Univ Penn Sch Vet Med Clin Studies Philadelphia PA 19104 USA;

Univ Penn Ophthalmol Philadelphia PA 19104 USA;

Univ Florida Ophthalmol Gainesville FL USA;

Univ Penn Sch Vet Med Clin Studies Philadelphia PA 19104 USA;

Univ Penn Sch Vet Med Clin Studies Philadelphia PA 19104 USA;

Univ Penn Ophthalmol Philadelphia PA 19104 USA;

Univ Penn Sch Vet Med Clin Studies Philadelphia PA 19104 USA;

Univ Florida Ophthalmol Gainesville FL USA;

Univ Penn Ophthalmol Philadelphia PA 19104 USA;

Univ Penn Sch Vet Med Clin Studies Philadelphia PA 19104 USA;

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正文语种 eng
中图分类眼科学 ;
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1. Long-term preservation of photoreceptor function and structure following early-stage treatment by AAV-mediated gene augmentation in canine model of NPHP5 Leber congenital amaurosis [J] . Aguirre Gustavo D., Cideciyan Artur V., Boye Sanford L., Investigative ophthalmology & visual science . 2018 ,第9期

机译：通过AAV介导的基因增强在NPHP5 Leber先天性神经症犬模型中的早期阶段治疗后的光感受器函数和结构的长期保存
2. AAV-mediated gene augmentation restores retinal function and vision in the canine model of NPHP5 Leber congenital amaurosis [J] . Aguirre Gustavo D., Cideciyan Artur V., Boye Sanford L., Investigative ophthalmology & visual science . 2016 ,第12期

机译：AAV介导的基因增强恢复NPHP5 Leber先天性生物症犬模型的视网膜功能和视觉
3. Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency. [J] . Mihelec M, Pearson RA, Robbie SJ, Human gene therapy . 2011 ,第10期

机译：基因治疗后由鸟苷酸环化酶-1缺乏引起的先天性黑ama病小鼠模型中的视锥细胞的长期保存和视觉功能的改善。
4. Analysis of Retinal Degeneration in a Leber Congenital Amaurosis Mouse Model Using High Spatial Resolution MALDI-Imaging Mass Spectrometry [C] . David M. G. Anderson, Zsolt Ablonczy, Yiannis Koutalos, ASMS Conference on Mass Spectrometry and Allied Topics . 2016

机译：利用高空间分辨率MATDI成像质谱法分析LEBER先天性生物疲劳小鼠模型的视网膜变性
5. The Leber Congenital Amaurosis CEP290 Cat Model: Working Towards a Cure. [D] . Minella, Andrea Louise. 2017

机译：Leber先天性无门诊CEP290猫模型：正在努力治愈。
6. Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency [O] . Marija Mihelec, Rachael A. Pearson, Scott J. Robbie, -1

机译：鸟苷酸环化酶-1缺乏引起的小鼠先天性无性先天性小鼠模型中基因治疗后锥体的长期保存和视觉功能的改善
7. Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency [O] . Mihelec, Marija, Pearson, Rachael A., Robbie, Scott J., 2011

机译：鸟苷酸环化酶-1缺乏引起的小鼠先天性无性先天性小鼠模型中基因治疗后锥体的长期保存和视觉功能的改善

Long-term preservation of photoreceptor function and structure following early-stage treatment by AAV-mediated gene augmentation in canine model of NPHP5 Leber congenital amaurosis

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