Antisense Oligonucleotide Therapies: ND

Antisense oligonucleotides are synthetic DNA oligomers that hybridize to a target RNA. This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases.

July 28, 2021

Evaluation of putative CSF biomarkers in paediatric spinal muscular atrophy (SMA) patients before and during treatment with nusinersen.

Journal of Cellular and Molecular Medicine
Jessika JohannsenJonas Denecke
July 5, 2021
Open Access

Generation, quality control, and analysis of the first genomically humanised knock-in mice for the ALS/FTD genes SOD1, TARDBP (TDP-43), and FUS

BioRxiv : the Preprint Server for Biology
A. DevoyThomas J Cunningham
July 3, 2021
Open Access

Advances of Antisense Oligonucleotide Technology in the Treatment of Hereditary Neurodegenerative Diseases.

Evidence-based Complementary and Alternative Medicine : ECAM
Mengsi LinJiaying Qiu
June 23, 2021

Translation of the poly(GR) frame in C9ORF72-ALS/FTD is regulated by cis-elements involved in alternative splicing.

Neurobiology of Aging
Alexa LampasonaFen-Biao Gao
June 12, 2021
Open Access

Survival motor neuron deficiency slows myoblast fusion through reduced myomaker and myomixer expression.

Journal of Cachexia, Sarcopenia and Muscle
Nikki M McCormackBarrington G Burnett
June 5, 2021

Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB.

Nature Neuroscience
Roy MaimonDon W Cleveland
June 2, 2021
Open Access

Increased Chitotriosidase1 Concentration Following Nusinersen Treatment in Spinal Muscular Atrophy

Maren FreigangRené Günther
May 21, 2021
Open Access

Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies.

Translational Neurodegeneration
Dunhui LiMay Thandar Aung-Htut
May 20, 2021

Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives.

Molecular Pharmaceutics
Gayathri R EdiriweeraKara L Vine
May 20, 2021
Open Access

Ataxin-2 gene: a powerful modulator of neurological disorders.

Current Opinion in Neurology
Jose Miguel Laffita-MesaPer Svenningsson
May 16, 2021

A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration.

Journal of Pharmacokinetics and Pharmacodynamics
Michael MonineIvan Nestorov
April 21, 2021
Open Access

Revisiting cell and gene therapies in Huntington's disease.

Journal of Neuroscience Research
Margarida BeatrizAna Cristina Carvalho Rego
April 16, 2021
Open Access

Reconditioning the Neurogenic Niche of Adult Non-human Primates by Antisense Oligonucleotide-Mediated Attenuation of TGFβ Signaling.

Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics
Sebastian PetersUlrich Bogdahn
April 16, 2021
Open Access

Quality of Life in SMA Patients Under Treatment With Nusinersen.

Frontiers in Neurology
Lucas MixDorothée Lulé
April 3, 2021

siRNA Therapeutics: Future Promise for Neurodegenerative Diseases.

Current Neuropharmacology
Atefeh AmiriAmirhossein Sahebkar
March 24, 2021
Open Access

Managing intrathecal administration of nusinersen in adolescents and adults with 5q-spinal muscular atrophy and previous spinal surgery.

Arquivos de neuro-psiquiatria
Rodrigo de Holanda MendonçaEdmar Zanoteli
March 18, 2021
Open Access

Mechanisms of repeat-associated non-AUG translation in neurological microsatellite expansion disorders.

Biochemical Society Transactions
Lydia M CastelliGuillaume M Hautbergue

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