Retinitis Pigmentosa Stem Cell Treatment

Stem Cell Treatmtent for Retinitis Pigmentosa

 
stem cell treatment for retinitis pigmentosa

Stem Cell Treatment for Retinitis Pigmentosa

Retinitis Pigmentosa treatments using stem cells is now an option...

Retinitis pigmentosa is a group of genetic eye conditions that leads to incurable blindness. In the progression of symptoms for Retinitis pigmentosa, night blindness generally precedes tunnel vision by years or even decades. Many people with Retinitis pigmentosa do not become legally blind until their 40s or 50s and retain some sight all their lives. Others go completely blind from Retinitis pigmentosa, in some cases as early as childhood. Progression of Retinitis pigmentosa is different in each case.

Retinitis pigmentosa is a type of progressive retinal dystrophy, a group of inherited disorders in which abnormalities of the photoreceptors (rods and cones) or the retinal pigment epithelium of the retina lead to progressive visual loss. Affected individuals first experience defective dark adaptation or nyctalopia (night blindness), followed by reduction of the peripheral visual field (known as tunnel vision) and, sometimes, loss of central vision late in the course of the disease.

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Stem Cell Treatment for Retinitis Pigmentosa

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Related Articles Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G. Proc Natl Acad Sci U S A. 2017 09 05;114(36):9695-9700 Authors: Emptoz A, Michel V, Lelli A, Akil O, Boutet de Monvel J, Lahlou G, Meyer A, Dupont T, Nouaille S, Ey E, Franca de Barros F, Beraneck M, Dulon D, Hardelin JP, Lustig L, Avan P, Petit C, Safieddine S Abstract Our understanding of the mechanisms underlying inherited forms of inner ear deficits has considerably improved during the past 20 y, but we are still far from curative treatments. We investigated gene replacement as a strategy for restoring inner ear functions in a mouse model of Usher syndrome type 1G, characterized by congenital profound deafness and balance disorders. These mice lack the scaffold protein sans, which is involved both in the morphogenesis of the stereociliary bundle, the sensory antenna of inner ear hair cells, and in the mechanoelectrical transduction process. We show that a single delivery of the sans cDNA by the adenoassociated virus 8 to the inner ear of newborn mutant mice reestablishes the expression and targeting of the protein to the tips of stereocilia. The therapeutic gene restores the architecture and mechanosensitivity of stereociliary bundles, improves hearing thresholds, and durably rescues these mice from the balance defects. Our results open up new perspectives for efficient gene therapy of cochlear and vestibular disorders by showing that even severe dysmorphogenesis of stereociliary bundles can be corrected. PMID: 28835534 [PubMed - indexed for MEDLINE]
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