glaucoma stem cell therapyRetinitis pigmentosa stem cell therapy

pubmed: retinitis pigmentosa...
NCBI: db=pubmed; Term=retinitis pigmentosa and stem cell treatment
NCBI pubmed
  • Reprogramming towards anabolism impedes degeneration in a preclinical model of retinitis pigmentosa.
    Related Articles

    Reprogramming towards anabolism impedes degeneration in a preclinical model of retinitis pigmentosa.

    Hum Mol Genet. 2016 Oct 01;25(19):4244-4255

    Authors: Zhang L, Justus S, Xu Y, Pluchenik T, Hsu CW, Yang J, Duong JK, Lin CS, Jia Y, Bassuk AG, Mahajan VB, Tsang SH

    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(H620Q/H620Q) preclinical RP mouse model and observed, functionally and morphologically, an improvement in the survival of rods and cones at early and late disease stages. These results elucidate the ability of reprogramming the metabolome to slow photoreceptor degeneration. This strategy may also be applicable to a wider range of neurodegenerative diseases, as enhancement of nutrient uptake is not gene-specific and is implicated in multiple pathologies. Enhancing anabolism promoted neuronal survival and function and could potentially benefit a number of photoreceptor and other degenerative conditions.

    PMID: 27516389 [PubMed - indexed for MEDLINE]

pubmed: stem cell treatment ...
NCBI: db=pubmed; Term=stem cell treatment and retinitis pigmentosa
NCBI pubmed
  • Reprogramming towards anabolism impedes degeneration in a preclinical model of retinitis pigmentosa.
    Related Articles

    Reprogramming towards anabolism impedes degeneration in a preclinical model of retinitis pigmentosa.

    Hum Mol Genet. 2016 Oct 01;25(19):4244-4255

    Authors: Zhang L, Justus S, Xu Y, Pluchenik T, Hsu CW, Yang J, Duong JK, Lin CS, Jia Y, Bassuk AG, Mahajan VB, Tsang SH

    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(H620Q/H620Q) preclinical RP mouse model and observed, functionally and morphologically, an improvement in the survival of rods and cones at early and late disease stages. These results elucidate the ability of reprogramming the metabolome to slow photoreceptor degeneration. This strategy may also be applicable to a wider range of neurodegenerative diseases, as enhancement of nutrient uptake is not gene-specific and is implicated in multiple pathologies. Enhancing anabolism promoted neuronal survival and function and could potentially benefit a number of photoreceptor and other degenerative conditions.

    PMID: 27516389 [PubMed - indexed for MEDLINE]