Antisense technology: an overview and prospectus
ST Crooke, BF Baker, RM Crooke… - Nature reviews Drug …, 2021 - nature.com
Antisense technology is now beginning to deliver on its promise to treat diseases by
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …
targeting RNA. Nine single-stranded antisense oligonucleotide (ASO) drugs representing …
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial
KA Strauss, MA Farrar, F Muntoni, K Saito… - Nature medicine, 2022 - nature.com
Abstract SPR1NT (NCT03505099) was a Phase III, multicenter, single-arm study to
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …
investigate the efficacy and safety of onasemnogene abeparvovec for presymptomatic …
Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
KA Strauss, MA Farrar, F Muntoni, K Saito… - Nature medicine, 2022 - nature.com
Most children with biallelic SMN1 deletions and three SMN2 copies develop spinal muscular
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …
atrophy (SMA) type 2. SPR1NT (NCT03505099), a Phase III, multicenter, single-arm trial …
RNA-binding proteins in human genetic disease
RNA-binding proteins (RBPs) are critical effectors of gene expression, and as such their
malfunction underlies the origin of many diseases. RBPs can recognize hundreds of …
malfunction underlies the origin of many diseases. RBPs can recognize hundreds of …
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 …
Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy
JR Mendell, SA Al-Zaidy, KJ Lehman, M McColly… - JAMA …, 2021 - jamanetwork.com
Importance This ongoing study assesses long-term safety and durability of response in
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …
infants with spinal muscular atrophy (SMA) type 1 after dosing with onasemnogene …
Antisense technology: A review
Antisense technology is beginning to deliver on the broad promise of the technology. Ten
RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double …
RNA-targeted drugs including eight single-strand antisense drugs (ASOs) and two double …
[HTML][HTML] Design of a randomized, placebo-controlled, phase 3 trial of tofersen initiated in clinically presymptomatic SOD1 variant carriers: the ATLAS study
M Benatar, J Wuu, PM Andersen, RC Bucelli… - …, 2022 - Elsevier
Despite extensive research, amyotrophic lateral sclerosis (ALS) remains a progressive and
invariably fatal neurodegenerative disease. Limited knowledge of the underlying causes of …
invariably fatal neurodegenerative disease. Limited knowledge of the underlying causes of …
Delivery of oligonucleotide therapeutics: chemical modifications, lipid nanoparticles, and extracellular vesicles
Oligonucleotides (ONs) comprise a rapidly growing class of therapeutics. In recent years, the
list of FDA-approved ON therapies has rapidly expanded. ONs are small (15–30 bp) …
list of FDA-approved ON therapies has rapidly expanded. ONs are small (15–30 bp) …