Journal article
Reprogramming towards anabolism impedes degeneration in a preclinical model of retinitis pigmentosa
Human molecular genetics, Vol.25(19), pp.4244-4255
10/01/2016
DOI: 10.1093/hmg/ddw256
PMCID: PMC5291198
PMID: 27516389
Abstract
Retinitis pigmentosa (RP) is an incurable neurodegenerative condition featuring photoreceptor death that leads to blindness. Currently, there is no approved therapeutic for photoreceptor degenerative conditions like RP and atrophic age-related macular degeneration (AMD). Although there are promising results in human gene therapy, RP is a genetically diverse disorder, such that gene-specific therapies would be practical in a small fraction of patients with RP. Here, we explore a non-gene-specific strategy that entails reprogramming photoreceptors towards anabolism by upregulating the mechanistic target of rapamycin (mTOR) pathway. We conditionally ablated the tuberous sclerosis complex 1 (Tsc1) gene, an mTOR inhibitor, in the rods of the Pde6b
Details
- Title: Subtitle
- Reprogramming towards anabolism impedes degeneration in a preclinical model of retinitis pigmentosa
- Creators
- Lijuan Zhang - Shanxi Eye Hospital, affiliated with Shanxi Medical University, Xinghualing, Taiyuan, Shanxi, ChinaSally Justus - Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USAYu Xu - Department of Ophthalmology, Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, ChinaTamara Pluchenik - Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USAChun-Wei Hsu - Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USAJin Yang - Tianjin Medical University Eye Hospital, Tianjin, ChinaJimmy K Duong - Department of Biostatistics, Mailman School of Public Health, Columbia University Medical Center, New York, USAChyuan-Sheng Lin - Department of Pathology and Cell Biology, Transgenic Animal Facility, Herbert Irving Comprehensive Cancer Center, College of Physicians and Surgeons of Columbia University, New York, NY, USAYading Jia - Shanxi Eye Hospital, affiliated with Shanxi Medical University, Xinghualing, Taiyuan, Shanxi, ChinaAlexander G Bassuk - Department of Pediatrics and Neurology, University of Iowa, Iowa City, IAVinit B Mahajan - Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, IA, USAStephen H Tsang - Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA
- Resource Type
- Journal article
- Publication Details
- Human molecular genetics, Vol.25(19), pp.4244-4255
- Publisher
- England
- DOI
- 10.1093/hmg/ddw256
- PMID
- 27516389
- PMCID
- PMC5291198
- ISSN
- 0964-6906
- eISSN
- 1460-2083
- Grant note
- R01 EY025225 / NEI NIH HHS R01 EY018213 / NEI NIH HHS R01 NS098590 / NINDS NIH HHS P30 CA013696 / NCI NIH HHS R01 EY024698 / NEI NIH HHS R01 EY026682 / NEI NIH HHS K08 EY020530 / NEI NIH HHS R01 EY024665 / NEI NIH HHS R21 AG050437 / NIA NIH HHS P30 EY019007 / NEI NIH HHS
- Language
- English
- Date published
- 10/01/2016
- Academic Unit
- Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
- Record Identifier
- 9984020794302771
Metrics
19 Record Views