July 31, 2026 preprint Open access A scalable platform for exon-skipping antisense oligonucleotide therapy development for inborn genetic diseases medRxiv DOI: 10.64898/2026.07.29.26359175 Full text (OA) OpenAlex Authors 0 Author list not loaded yet. Cited by 0 stored of 0 References 11 Search Sort Most cited Newest Oldest Patent citations Title Any typearticle review book-chapter conference-paper preprint dissertation book dataset other Any fieldAgricultural and Biological Sciences Arts and Humanities Biochemistry, Genetics and Molecular Biology Business, Management and Accounting Chemical Engineering Chemistry Computer Science Decision Sciences Dentistry Earth and Planetary Sciences Economics, Econometrics and Finance Energy Engineering Environmental Science Health Professions Immunology and Microbiology Materials Science Mathematics Medicine Neuroscience Nursing Pharmacology, Toxicology and Pharmaceutics Physics and Astronomy Psychology Social Sciences Veterinary Open access Delivery of oligonucleotide‐based therapeutics: challenges and opportunities 2021 EMBO Molecular Medicine article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 13 patents 388 citations Possibilities and limitations of antisense oligonucleotide therapies for the treatment of monogenic disorders 2024 Communications Medicine article Biochemistry, Genetics and Molecular Biology RNA Interference and Gene Delivery Open access cited by 5 patents 272 citations The LOVD3 platform: efficient genome-wide sharing of genetic variants 2021 European Journal of Human Genetics article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access 157 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 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 Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides 2022 Nucleic Acid Therapeutics article Biochemistry, Genetics and Molecular Biology RNA Research and Splicing Open access 55 citations Consensus guidelines for assessing eligibility of pathogenic DNA variants for antisense oligonucleotide treatments 2025 The American Journal of Human Genetics article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases Open access 32 citations Stargardt disease-associated in-frame ABCA4 exon 17 skipping results in significant ABCA4 function 2023 Journal of Translational Medicine article Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders Open access 10 citations Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study 2025 Genetics in Medicine article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases 4 citations Interventional genomics: Bridging germline diagnosis and therapeutic action 2026 Genetics in Medicine article Biochemistry, Genetics and Molecular Biology Genomics and Rare Diseases 3 citations Expanding the options for therapeutic exon skipping as a future treatment for USH2A -associated disease by 3D structural modeling of newly formed hybrid domains 2026 bioRxiv (Cold Spring Harbor Laboratory) preprint Biochemistry, Genetics and Molecular Biology Retinal Development and Disorders 1 citations 11 results