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

Behavioral deficits in an Angelman syndrome model: Effects of genetic background and age

(2013) Hsien-Sung Huang et al.   BEHAVIOURAL BRAIN RESEARCH

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

(2013) Timothy M Miller et al.   LANCET NEUROLOGY

Topoisomerases facilitate transcription of long genes linked to autism

(2013) Ian F. King et al.   NATURE

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

(2013) C. Lagier-Tourenne et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Truncation of Ube3a-ATS Unsilences Paternal Ube3a and Ameliorates Behavioral Defects in the Angelman Syndrome Mouse Model

(2013) Linyan Meng et al.   PLoS Genetics

Prader-Willi syndrome

(2012) Suzanne B. Cassidy et al.   GENETICS IN MEDICINE

Ube3a-ATS is an atypical RNA polymerase II transcript that represses the paternal expression of Ube3a

(2012) Linyan Meng et al.   HUMAN MOLECULAR GENETICS

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

(2012) Holly B. Kordasiewicz et al.   NEURON

Normal social seeking behavior, hypoactivity and reduced exploratory range in a mouse model of Angelman syndrome

(2011) Melody Allensworth et al.   BMC GENETICS

Topoisomerase inhibitors unsilence the dormant allele of Ube3a in neurons

(2011) Hsien-Sung Huang et al.   NATURE

Clinical and genetic aspects of Angelman syndrome

(2010) Charles A Williams et al.   GENETICS IN MEDICINE