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About 125 results

ALLMedicine™ Crystalline Dystrophy Center

Research & Reviews  46 results

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
https://clinicaltrials.gov/ct2/show/NCT05399069

Jun 1st, 2022 - VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo 365(±7) days of safety observation ...

Accumulation of Lipid Droplets in a Novel Bietti Crystalline Dystrophy Zebrafish Model ...
https://doi.org/10.1167/iovs.63.5.32
Investigative Ophthalmology & Visual Science; Gao P, Jia D et. al.

May 27th, 2022 - Bietti crystalline dystrophy (BCD) is a progressive retinal degenerative disease primarily characterized by numerous crystal-like deposits and degeneration of retinal pigment epithelium (RPE) and photoreceptor cells. CYP4V2 (cytochrome P450 family...

A novel and efficient murine model of Bietti crystalline dystrophy.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8906172
Disease Models & Mechanisms; Wang Y, Liu Y et. al.

Mar 2nd, 2022 - Bietti crystalline dystrophy (BCD) is an autosomal recessive inherited retinal disease, resulting in blindness in most patients. The etiology and development mechanism of it remain unclear. Given the defects in previous mouse models of BCD, we gen...

PREDICTED PROTEIN STRUCTURE VARIATIONS INDICATE THE CLINICAL PRESENTATION OF CYP4V2-REL...
https://doi.org/10.1097/IAE.0000000000003381
Retina (Philadelphia, Pa.); Chan LW, Sung YC et. al.

Dec 20th, 2021 - To investigate the relationship between different CYP4V2 disease-causing variants and disease severity in Bietti crystalline dystrophy (BCD). Twenty-one subjects from 19 unrelated families with a clinical diagnosis of BCD were enrolled. A novel se...

A convenient test system for the identification of CYP4V2 inhibitors.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8598247
Molecular Vision; Sharma S, Liu S et. al.

Dec 10th, 2021 - Polymorphisms in the gene that codes for the human cytochrome P450 enzyme CYP4V2 are a cause of Bietti crystalline dystrophy (BCD). Therefore, inhibition of CYP4V2 activity may well be a cause of visual disability. However, monitoring the fatty ac...

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Clinicaltrials.gov  4 results

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
https://clinicaltrials.gov/ct2/show/NCT05399069

Jun 1st, 2022 - VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo 365(±7) days of safety observation ...

The Gene Replacement Therapy by rAAV2/8-hCYP4V2
https://clinicaltrials.gov/ct2/show/NCT04722107

May 10th, 2021 - A Safety Study of The Gene Replacement Therapy by rAAV2/8-hCYP4V2 Following a Single Subretinal Administration in The Patients of Bietti's Crystalline Dystrophy (BCD)

Management of Retinitis Pigmentosa by Mesenchymal Stem Cells by Wharton's Jelly Derived Mesenchymal Stem Cells
https://clinicaltrials.gov/ct2/show/NCT04224207

Jan 13th, 2020 - The retinal pigment epithelium (RPE) forms the outer blood-retinal barrier between photoreceptor cells and choroidal blood vessels. Photoreceptor cells are vitally and functionally dependent on the RPE. The conversion of blood glucose to ATP, synt...

Molecular Genetics of Retinal Degenerations
https://clinicaltrials.gov/ct2/show/NCT00231010

Apr 5th, 2018 - Objective: This project, Molecular Genetics of Retinal Degenerations will study the inheritance of genetic retinal degenerations, both Mendelian and complex, in families of many nationalities and ethnic backgrounds in order to identify the genes t...

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