Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases
Sign Up to like & getrecommendations! Published in 2020 at "Expert Opinion on Investigational Drugs"
DOI: 10.1080/13543784.2020.1804853
Abstract: ABSTRACT Introduction Antisense oligonucleotides (ASOs) represent a class of drugs which can be rationally designed to complement the coding or non-coding regions of target RNA transcripts. They could modulate pre-messenger RNA splicing, induce mRNA knockdown,… read more here.
Keywords: clinical trials; antisense oligonucleotide; inherited retinal; treatment inherited ... See more keywords
Treatment of inherited thrombocytopenias
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DOI: 10.3324/haematol.2022.280856
Abstract: The new techniques of genetic analysis have made it possible to identify many new forms of inherited thrombocytopenias (IT) and study large series of patients. In recent years, this has changed the view of IT,… read more here.
Keywords: inherited thrombocytopenias; treatment inherited;
Management and treatment of inherited retinal dystrophies
Sign Up to like & getrecommendations! Published in 2021 at "Taiwan Journal of Ophthalmology"
DOI: 10.4103/tjo.tjo_32_21
Abstract: I retinal dystrophies (IRDs) are a rare group of hereditary diseases that lead to progressive degeneration of retinal cells.[1] While there are currently several ongoing clinical trials utilizing pharmacological agents and adeno‐associated virus (AAV) vector‐mediated… read more here.
Keywords: treatment; retinal dystrophies; management treatment; inherited retinal ... See more keywords