Non-coding RNAs in development and disease: background, mechanisms, and therapeutic approaches

J Beermann, MT Piccoli, J Viereck… - Physiological …, 2016 - journals.physiology.org
Advances in RNA-sequencing techniques have led to the discovery of thousands of non-
coding transcripts with unknown function. There are several types of non-coding linear …

Transcription regulation by long non-coding RNAs: mechanisms and disease relevance

J Ferrer, N Dimitrova - Nature Reviews Molecular Cell Biology, 2024 - nature.com
Long non-coding RNAs (lncRNAs) outnumber protein-coding transcripts, but their functions
remain largely unknown. In this Review, we discuss the emerging roles of lncRNAs in the …

Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA

JM Wolter, H Mao, G Fragola, JM Simon, JL Krantz… - Nature, 2020 - nature.com
Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by a mutation or
deletion of the maternally inherited UBE3A allele. In neurons, the paternally inherited …

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA

L Meng, AJ Ward, S Chun, CF Bennett, AL Beaudet… - Nature, 2015 - nature.com
Angelman syndrome is a single-gene disorder characterized by intellectual disability,
developmental delay, behavioural uniqueness, speech impairment, seizures and ataxia,. It …

Angelman syndrome—insights into a rare neurogenetic disorder

K Buiting, C Williams, B Horsthemke - Nature Reviews Neurology, 2016 - nature.com
Angelman syndrome is a rare neurogenetic disorder that is characterized by microcephaly,
severe intellectual deficit, speech impairment, epilepsy, EEG abnormalities, ataxic …

Long non-coding RNA (lncRNA) roles in cell biology, neurodevelopment and neurological disorders

V Aliperti, J Skonieczna, A Cerase - Non-coding RNA, 2021 - mdpi.com
Development is a complex process regulated both by genetic and epigenetic and
environmental clues. Recently, long non-coding RNAs (lncRNAs) have emerged as key …

Computational models for lncRNA function prediction and functional similarity calculation

X Chen, YZ Sun, NN Guan, J Qu… - Briefings in …, 2019 - academic.oup.com
From transcriptional noise to dark matter of biology, the rapidly changing view of long non-
coding RNA (lncRNA) leads to deep understanding of human complex diseases induced by …

Ube3a reinstatement identifies distinct developmental windows in a murine Angelman syndrome model

S Silva-Santos, GM Van Woerden… - The Journal of …, 2015 - Am Soc Clin Investig
Angelman syndrome (AS) is a severe neurodevelopmental disorder that results from loss of
function of the maternal ubiquitin protein ligase E3A (UBE3A) allele. Due to neuron-specific …

Long noncoding RNAs: Lessons from genomic imprinting

C Kanduri - Biochimica et Biophysica Acta (BBA)-Gene Regulatory …, 2016 - Elsevier
Genomic imprinting has been a great resource for studying transcriptional and post-
transcriptional-based gene regulation by long noncoding RNAs (lncRNAs). In this article, I …

An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript

SV Dindot, S Christian, WJ Murphy, A Berent… - Science Translational …, 2023 - science.org
Angelman syndrome is a devastating neurogenetic disorder for which there is currently no
effective treatment. It is caused by mutations or epimutations affecting the expression or …