AAV5-miHTT gene therapy demonstrates broad distribution and strong human mutant huntingtin lowering in a Huntington's disease minipig model
MM Evers, J Miniarikova, S Juhas, A Vallès… - Molecular Therapy, 2018 - cell.com
Huntington's disease (HD) is a fatal neurodegenerative disorder caused by a CAG
trinucleotide repeat expansion in the huntingtin gene. Previously, we showed strong …
trinucleotide repeat expansion in the huntingtin gene. Previously, we showed strong …
Translation of microRNA-based huntingtin-lowering therapies from preclinical studies to the clinic
J Miniarikova, MM Evers, P Konstantinova - Molecular Therapy, 2018 - cell.com
The single mutation underlying the fatal neuropathology of Huntington's disease (HD) is a
CAG triplet expansion in exon 1 of the huntingtin (HTT) gene, given rise to a toxic mutant …
CAG triplet expansion in exon 1 of the huntingtin (HTT) gene, given rise to a toxic mutant …
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
RNA interference (RNAi) for spinocerebellar ataxia type 1 can prevent and reverse
behavioral deficits and neuropathological readouts in mouse models, with safety and benefit …
behavioral deficits and neuropathological readouts in mouse models, with safety and benefit …
Design, characterization, and lead selection of therapeutic miRNAs targeting huntingtin for development of gene therapy for Huntington's disease
J Miniarikova, I Zanella, A Huseinovic… - … Therapy-Nucleic Acids, 2016 - cell.com
Huntington's disease (HD) is a neurodegenerative disorder caused by accumulation of CAG
expansions in the huntingtin (HTT) gene. Hence, decreasing the expression of mutated HTT …
expansions in the huntingtin (HTT) gene. Hence, decreasing the expression of mutated HTT …
Recent advances in molecular therapies for neurological disease: triplet repeat disorders
P Gonzalez-Alegre - Human Molecular Genetics, 2019 - academic.oup.com
Triplet repeat diseases (TRDs) are caused by pathogenic expansions of trinucleotide
sequence repeats within coding and non-coding regions of different genes. They are …
sequence repeats within coding and non-coding regions of different genes. They are …
Chiral control
D Butler, N Iwamoto, N Svrzikapa, GL Verdine… - US Patent …, 2018 - Google Patents
The present invention relates to chirally controlled oligonucleotides, chirally controlled
oligonucleotide compositions, and the method of making and using the same. The invention …
oligonucleotide compositions, and the method of making and using the same. The invention …
Methods for the synthesis of functionalized nucleic acids
GL Verdine, N Iwamoto, DCD Butler - US Patent 9,605,019, 2017 - Google Patents
The present application, among other things, provides technologies, eg, reagents, methods,
etc. for preparing oligonucleotides comprising phosphorothiotriesters linkages, eg …
etc. for preparing oligonucleotides comprising phosphorothiotriesters linkages, eg …
Method for the synthesis of phosphorus atom modified nucleic acids
T Wada, M Shimizu - US Patent 9,695,211, 2017 - Google Patents
US9695211B2 - Method for the synthesis of phosphorus atom modified nucleic acids - Google
Patents US9695211B2 - Method for the synthesis of phosphorus atom modified nucleic acids …
Patents US9695211B2 - Method for the synthesis of phosphorus atom modified nucleic acids …
A novel system for glycosylation engineering by natural and artificial miRNAs
F Klingler, L Naumann, P Schlossbauer, L Dreyer… - Metabolic …, 2023 - Elsevier
N-linked glycosylation is a crucial post-translational modification of many
biopharmaceuticals, including monoclonal antibodies (mAbs), capable of modifying their …
biopharmaceuticals, including monoclonal antibodies (mAbs), capable of modifying their …
The long non-coding RNA NEAT1 is elevated in polyglutamine repeat expansion diseases and protects from disease gene-dependent toxicities
C Cheng, RM Spengler, MS Keiser… - Human molecular …, 2018 - academic.oup.com
Polyglutamine (polyQ) repeat diseases are a class of neurodegenerative disorders caused
by CAG-repeat expansion. There are diverse cellular mechanisms behind the pathogenesis …
by CAG-repeat expansion. There are diverse cellular mechanisms behind the pathogenesis …