Overcoming cellular barriers for RNA therapeutics
SF Dowdy - Nature biotechnology, 2017 - nature.com
RNA-based therapeutics, such as small-interfering (siRNAs), microRNAs (miRNAs),
antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR–Cas9, have …
antisense oligonucleotides (ASOs), aptamers, synthetic mRNAs and CRISPR–Cas9, have …
Non-coding RNAs as drug targets
M Matsui, DR Corey - Nature reviews Drug discovery, 2017 - nature.com
Most of the human genome encodes RNAs that do not code for proteins. These non-coding
RNAs (ncRNAs) may affect normal gene expression and disease progression, making them …
RNAs (ncRNAs) may affect normal gene expression and disease progression, making them …
Single-dose gene-replacement therapy for spinal muscular atrophy
JR Mendell, S Al-Zaidy, R Shell… - … England Journal of …, 2017 - Mass Medical Soc
Background Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor
neuron disease with an onset during infancy that results in failure to achieve motor …
neuron disease with an onset during infancy that results in failure to achieve motor …
Nusinersen versus sham control in later-onset spinal muscular atrophy
Background Nusinersen is an antisense oligonucleotide drug that modulates pre–
messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been …
messenger RNA splicing of the survival motor neuron 2 (SMN2) gene. It has been …
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Background Nusinersen is a 2′-O-methoxyethyl phosphorothioate-modified antisense drug
being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to …
being developed to treat spinal muscular atrophy. Nusinersen is specifically designed to …
Base editing rescue of spinal muscular atrophy in cells and in mice
Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …
Mechanisms and regulation of alternative pre-mRNA splicing
Y Lee, DC Rio - Annual review of biochemistry, 2015 - annualreviews.org
Precursor messenger RNA (pre-mRNA) splicing is a critical step in the posttranscriptional
regulation of gene expression, providing significant expansion of the functional proteome of …
regulation of gene expression, providing significant expansion of the functional proteome of …
Antisense oligonucleotides: the next frontier for treatment of neurological disorders
C Rinaldi, MJA Wood - Nature Reviews Neurology, 2018 - nature.com
Antisense oligonucleotides (ASOs) were first discovered to influence RNA processing and
modulate protein expression over two decades ago; however, progress translating these …
modulate protein expression over two decades ago; however, progress translating these …
[HTML][HTML] Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE …
Spinal muscular atrophy (SMA) is a neurodegenerative disease associated with severe
muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety …
muscle atrophy and weakness in the limbs and trunk. We report interim efficacy and safety …
Recent progress in non-native nucleic acid modifications
While Nature harnesses RNA and DNA to store, read and write genetic information, the
inherent programmability, synthetic accessibility and wide functionality of these nucleic acids …
inherent programmability, synthetic accessibility and wide functionality of these nucleic acids …