Emerging Concepts for RNA Therapeutics for Inherited Retinal Disease
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Publisher
Springer International Publishing
Link
http://link.springer.com/content/pdf/10.1007/978-3-030-27378-1_14
Reference32 articles.
1. Adams D, Gonzalez-Duarte A, O’Riordan WD et al (2018) Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med 379:11–21
2. Albert S, Garanto A, Sangermano R et al (2018) Identification and rescue of splice defects caused by two neighboring deep-Intronic ABCA4 mutations underlying Stargardt disease. Am J Hum Genet 102:517–527
3. Bonifert T, Gonzalez Menendez I, Battke F et al (2016) Antisense oligonucleotide mediated splice correction of a deep Intronic mutation in OPA1. Mol Ther Nucleic Acids 5:e390
4. Chadderton N, Millington-Ward S, Palfi A et al (2009) Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy. Mol Ther 17:593–599
5. Chan L, Mahajan VB, Tsang SH (2017) Genome surgery and gene therapy in retinal disorders. Yale J Biol Med 90:523–532
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