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Advances in oligonucleotide drug delivery
TC Roberts, R Langer, MJA Wood - Nature reviews Drug discovery, 2020 - nature.com
Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …
Protein aggregation and neurodegenerative disease
CA Ross, MA Poirier - Nature medicine, 2004 - nature.com
Neurodegenerative diseases such as Alzheimer's disease (AD), Parkinson's disease (PD),
Huntington's disease (HD), amyotrophic lateral sclerosis (ALS) and prion diseases are …
Huntington's disease (HD), amyotrophic lateral sclerosis (ALS) and prion diseases are …
Small silencing RNAs: an expanding universe
M Ghildiyal, PD Zamore - Nature reviews genetics, 2009 - nature.com
Since the discovery in 1993 of the first small silencing RNA, a dizzying number of small RNA
classes have been identified, including microRNAs (miRNAs), small interfering RNAs …
classes have been identified, including microRNAs (miRNAs), small interfering RNAs …
Inherited retinal diseases: linking genes, disease-causing variants, and relevant therapeutic modalities
N Schneider, Y Sundaresan, P Gopalakrishnan… - Progress in retinal and …, 2022 - Elsevier
Inherited retinal diseases (IRDs) are a clinically complex and heterogenous group of visual
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …
RNAi therapeutics: principles, prospects and challenges
RNA interference (RNAi) was discovered less than a decade ago and already there are
human clinical trials in progress or planned. A major advantage of RNAi versus other …
human clinical trials in progress or planned. A major advantage of RNAi versus other …
Unlocking the potential of the human genome with RNA interference
The discovery of RNA interference (RNAi) may well be one of the transforming events in
biology in the past decade. RNAi can result in gene silencing or even in the expulsion of …
biology in the past decade. RNAi can result in gene silencing or even in the expulsion of …
siRNAs: applications in functional genomics and potential as therapeutics
Y Dorsett, T Tuschl - Nature reviews Drug discovery, 2004 - nature.com
Molecules that can specifically silence gene expression are powerful research tools. Much
effort has been put into the development of such molecules and has resulted in the creation …
effort has been put into the development of such molecules and has resulted in the creation …
Functional and hyperfunctional siRNA
A Khvorova, A Reynolds, D Leake, W Marshall… - US Patent …, 2012 - Google Patents
US8090542B2 - Functional and hyperfunctional siRNA - Google Patents US8090542B2 -
Functional and hyperfunctional siRNA - Google Patents Functional and hyperfunctional …
Functional and hyperfunctional siRNA - Google Patents Functional and hyperfunctional …
MicroRNA control of PHABULOSA in leaf development: importance of pairing to the microRNA 5′ region
AC Mallory, BJ Reinhart, MW Jones‐Rhoades… - The EMBO …, 2004 - embopress.org
MicroRNAs (miRNAs) are∼ 22‐nucleotide noncoding RNAs that can regulate gene
expression by directing mRNA degradation or inhibiting productive translation. Dominant …
expression by directing mRNA degradation or inhibiting productive translation. Dominant …
Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi
Huntington's disease (HD) is a fatal, dominant neurodegenerative disease caused by a
polyglutamine repeat expansion in exon 1 of the HD gene, which encodes the huntingtin …
polyglutamine repeat expansion in exon 1 of the HD gene, which encodes the huntingtin …