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ProQR Therapeutics (Netherlands) Sign in to save

47 stored papers · Search its papers → · Its authors →

Papers 47

The Critical Contribution of Pseudouridine to mRNA COVID-19 Vaccines
2021 Frontiers in Cell and Developmental Biology article Medicine SARS-CoV-2 and COVID-19 Research Open access cited by 63 patents

274

citations
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations
2021 Molecular Therapy article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 8 patents

148

citations
Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b/2 trial
2022 Nature Medicine article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

134

citations
Spliceosomal snRNA Epitranscriptomics
2021 Frontiers in Genetics article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access cited by 1 patent

116

citations
From Antisense RNA to RNA Modification: Therapeutic Potential of RNA-Based Technologies
2021 Biomedicines article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 3 patents

81

citations
Targeted pseudouridylation: An approach for suppressing nonsense mutations in disease genes
2023 Molecular Cell article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access cited by 3 patents

70

citations
Suppression of Nonsense Mutations by New Emerging Technologies
2020 International Journal of Molecular Sciences article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access

67

citations
Durable vision improvement after a single treatment with antisense oligonucleotide sepofarsen: a case report
2021 Nature Medicine article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 1 patent

64

citations
LEBER CONGENITAL AMAUROSIS DUE TO CEP290 MUTATIONS—SEVERE VISION IMPAIRMENT WITH A HIGH UNMET MEDICAL NEED
2021 Retina article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 1 patent

61

citations
QR-313, an Antisense Oligonucleotide, Shows Therapeutic Efficacy for Treatment of Dominant and Recessive Dystrophic Epidermolysis Bullosa: A Preclinical Study
2020 Journal of Investigative Dermatology article Biochemistry, Genetics and Molecular Biology Skin and Cellular Biology Research Open access

56

citations
Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C
2023 Molecular Therapy — Nucleic Acids article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

41

citations
Distinct contribution of cone photoreceptor subtypes to the mammalian biological clock
2021 Proceedings of the National Academy of Sciences article Neuroscience Circadian rhythm and melatonin Open access

36

citations
RNA-based therapies in inherited retinal diseases
2022 Therapeutic Advances in Ophthalmology article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access cited by 1 patent

36

citations
Rational Design of RNA Editing Guide Strands: Cytidine Analogs at the Orphan Position
2021 Journal of the American Chemical Society article Biochemistry, Genetics and Molecular Biology RNA regulation and disease Open access cited by 27 patents

32

citations
Improving Translation by Identifying Evidence for More Human-Relevant Preclinical Strategies
2020 Animals article Veterinary Animal testing and alternatives Open access

28

citations
Restoration of Cone Sensitivity to Individuals with Congenital Photoreceptor Blindness within the Phase 1/2 Sepofarsen Trial
2022 Ophthalmology Science article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

18

citations
Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes
2023 American Journal of Ophthalmology Case Reports article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

17

citations
A pathogenic proteolysis–resistant huntingtin isoform induced by an antisense oligonucleotide maintains huntingtin function
2022 JCI Insight article Neuroscience Genetic Neurodegenerative Diseases Open access

14

citations
QR‐421a RNA therapy in retinitis pigmentosa due to mutations in USH2A: Stellar trial Phase[AP1] 1b/2 interim results
2022 Acta Ophthalmologica article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders

12

citations
Stargardt disease-associated missense and synonymous ABCA4 variants result in aberrant splicing
2023 Human Molecular Genetics article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access

10

citations

Most cited authors