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Artur V Cideciyan Selected Research

Leber Congenital Amaurosis

1/2022Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial.
2/2021Leber Congenital Amaurosis Due to GUCY2D Mutations: Longitudinal Analysis of Retinal Structure and Visual Function.
1/2021Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report.
1/2020Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy.
1/2019Treatment Potential for Macular Cone Vision in Leber Congenital Amaurosis Due to CEP290 or NPHP5 Mutations: Predictions From Artificial Intelligence.
1/2019Effect of an intravitreal antisense oligonucleotide on vision in Leber congenital amaurosis due to a photoreceptor cilium defect.
11/2017In Vitro Modeling Using Ciliopathy-Patient-Derived Cells Reveals Distinct Cilia Dysfunctions Caused by CEP290 Mutations.
1/2017Postretinal Structure and Function in Severe Congenital Photoreceptor Blindness Caused by Mutations in the GUCY2D Gene.
10/2016Overlap of abnormal photoreceptor development and progressive degeneration in Leber congenital amaurosis caused by NPHP5 mutation.
1/2016Leber Congenital Amaurosis: Genotypes and Retinal Structure Phenotypes.
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Artur V Cideciyan Research Topics

Disease

28Leber Congenital Amaurosis
01/2022 - 04/2004
24Retinitis Pigmentosa (Pigmentary Retinopathy)
01/2021 - 04/2002
21Retinal Degeneration
01/2019 - 04/2002
18Blindness (Hysterical Blindness)
01/2020 - 04/2002
6Ciliopathies
01/2021 - 04/2011
6Retinal Diseases
01/2020 - 02/2004
5Blue cone monochromatism
01/2022 - 12/2013
5Stargardt Disease
10/2021 - 03/2004
4Macular Degeneration (Age-Related Maculopathy)
01/2017 - 07/2002
3Atrophy
01/2020 - 05/2015
3Cone-Rod Dystrophies
01/2019 - 06/2002
3Disease Progression
07/2017 - 05/2009
3Choroideremia
01/2016 - 03/2002
3Vision Disorders (Hemeralopia)
12/2013 - 04/2004
2Low Vision (Subnormal Vision)
01/2022 - 01/2016
2Photophobia (Light Sensitivity)
01/2021 - 01/2017
2Scotoma (Scintillating Scotoma)
01/2017 - 04/2008
2Diabetic Retinopathy (Retinopathy, Diabetic)
01/2017 - 04/2005
2Usher Syndromes (Usher Syndrome)
01/2016 - 06/2008
2Laminopathies
03/2014 - 04/2008
2Enhanced S-Cone Syndrome
03/2003 - 01/2002
1Inborn Genetic Diseases (Disease, Hereditary)
01/2021
1Toxic Optic Neuropathy
01/2020
1Agenesis of Cerebellar Vermis
11/2017
1Geographic Atrophy
04/2015
1type 1 Amaurosis congenita of Leber
01/2013
1Type Ib Usher Syndrome
10/2011
1type 1B Usher syndrome
04/2009
1Bardet-Biedl Syndrome (Syndrome, Bardet-Biedl)
11/2006
1Hypertrophy
09/2006
1Retinal Perforations
08/2006
1Choroidal Neovascularization
04/2005
1Macular Edema
04/2005
1Late-Onset Retinal Degeneration
10/2003
1Vitelliform Macular Dystrophy
02/2003
1Dog Diseases (Disease, Canine)
04/2002

Drug/Important Bio-Agent (IBA)

56Retinaldehyde (Retinal)IBA
01/2022 - 01/2002
12Proteins (Proteins, Gene)FDA Link
01/2019 - 09/2006
7GTP Phosphohydrolases (GTPases)IBA
01/2021 - 05/2007
7RetinoidsIBA
01/2017 - 04/2005
6Rhodopsin (Visual Purple)IBA
10/2019 - 04/2002
3Antisense OligonucleotidesIBA
01/2022 - 01/2019
2Guanylate Cyclase (Guanylyl Cyclase)IBA
01/2019 - 01/2013
2Myosins (Myosin)IBA
01/2016 - 10/2011
2guanylate cyclase 1IBA
08/2015 - 06/2006
2LipofuscinIBA
06/2015 - 05/2015
2retinoid isomerohydrolase (retinol isomerase)IBA
06/2009 - 09/2007
2LuteinIBA
03/2007 - 03/2002
2Macular PigmentIBA
03/2007 - 03/2002
1Protein Isoforms (Isoforms)IBA
01/2021
1Biomarkers (Surrogate Marker)IBA
01/2020
1N- (4- azidobenzoylglycyl)- S- (2- thiopyridyl)cysteine (AGTC)IBA
01/2020
1Codon (Codons)IBA
01/2020
1CalciumIBA
01/2019
1Adenosine Triphosphate (ATP)IBA
01/2017
1hydroxide ionIBA
01/2017
1Retinal Pigments (Pigments, Visual)IBA
01/2017
1Complement System Proteins (Complement)IBA
01/2016
1Cone OpsinsIBA
01/2015
1WD repeat containing planar cell polarity effectorIBA
04/2012
1Phosphotransferases (Kinase)IBA
12/2011
1OpsinsIBA
03/2011
1dehydrodolichyl diphosphate synthetaseIBA
02/2011
1IsomerasesIBA
12/2010
1lecithin-retinol acyltransferaseIBA
06/2009
1DNA (Deoxyribonucleic Acid)IBA
07/2008
1Melanins (Melanin)IBA
04/2008
1Complementary DNA (cDNA)IBA
04/2005
1Vascular Endothelial Growth Factor A (Vascular Endothelial Growth Factor)IBA
04/2005
1ATP-Binding Cassette Transporters (ABC Transporters)IBA
03/2004
1CollagenIBA
10/2003
1G-Protein-Coupled Receptor Kinase 1 (G Protein-Coupled Receptor Kinase 1)IBA
03/2003
1Chloride Channels (Chloride Channel)IBA
02/2003
1Ataxin-7IBA
06/2002
1Transcription Factors (Transcription Factor)IBA
06/2002

Therapy/Procedure

19Therapeutics
02/2021 - 02/2005
2Visual Prosthesis
10/2016 - 04/2012
1Precision Medicine
01/2020
1Intravitreal Injections
01/2019
1Injections
08/2006