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Intrastriatal administration of AAV5-miHTT in non-human primates and rats is well tolerated and results in miHTT transgene expression in key areas of Huntington disease pathology
Cerebral organoids
AAV5-miHTT lowers huntingtin mRNA and protein without off-target effects in patient-derived neuronal cultures and astrocytes
Development of an AAV-based microRNA gene therapy to treat Machado-Joseph disease
AAV5-miHTT gene therapy demonstrates sustained Huntingtin lowering and functional improvement in Huntington disease mouse models
Targeting RNA-mediated toxicity in C9orf72 ALS and/or FTD by RNAi-based gene therapy
Artificial microRNAs targeting C9orf72 can reduce accumulation of intra-nuclear transcripts in ALS and FTD patients
Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model
Transcriptional profiling and biomarker identification reveal tissue specific effects of expanded ataxin-3 in a spinocerebellar ataxia type 3 mouse model
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington's disease
In vivo proof-of-concept of removal of the huntingtin caspase cleavage motif-encoding exon 12 approach in the YAC128 mouse model of Huntington's disease
Antisense oligonucleotides in therapy for neurodegenerative disorders
Making (anti-) sense out of huntingtin levels in Huntington disease
Developing genetic therapies for polyglutamine disorders
Preventing Formation of Toxic N-Terminal Huntingtin Fragments Through Antisense Oligonucleotide-Mediated Protein Modification
Ataxin-3 protein modification as a treatment strategy for spinocerebellar ataxia type 3: Removal of the CAG containing exon
REDUCING TOXIC N-TERMINAL HUNTINGTIN FRAGMENTS IN HD USING EXON SKIPPING
Antisense-mediated RNA targeting: versatile and expedient genetic manipulation in the brain
Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide