AAV2/4-RS1 gene therapy in the retinoschisin knockout mouse model of X-linked retinoschisis
Sign Up to like & getrecommendations! Published in 2022 at "PLOS ONE"
DOI: 10.1371/journal.pone.0276298
Abstract: Objective To evaluate efficacy of a novel adeno-associated virus (AAV) vector, AAV2/4-RS1, for retinal rescue in the retinoschisin knockout (Rs1-KO) mouse model of X-linked retinoschisis (XLRS). Brinzolamide (Azopt®), a carbonic anhydrase inhibitor, was tested for… read more here.
Keywords: aav2 rs1; rs1; retinoschisin knockout; mice ... See more keywords
Effects of Recombinant Spidroin rS1/9 on Brain Neural Progenitors After Photothrombosis-Induced Ischemia
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DOI: 10.3389/fcell.2020.00823
Abstract: The existence of niches of stem cells residence in the ventricular–subventricular zone and the subgranular zone in the adult brain is well-known. These zones are the sites of restoration of brain function after injury. Bioengineered… read more here.
Keywords: photothrombosis induced; spidroin rs1; progenitor cells; brain ... See more keywords
Predominant Founder Effect among Recurrent Pathogenic Variants for an X-Linked Disorder
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DOI: 10.3390/genes13040675
Abstract: For disorders with X-linked inheritance, variants may be transmitted through multiple generations of carrier females before an affected male is ascertained. Pathogenic RS1 variants exclusively cause X-linked retinoschisis (XLRS). While RS1 is constrained to variation,… read more here.
Keywords: recurrent pathogenic; founder effect; founder; rs1 ... See more keywords