Abstract:
This application relates to a method for converting somatic cells to Neural Stem Cells (NSCs). Moreover this application relates to a method for converting human fibroblasts, keratinocytes or adipocytes to neural stem cells based on linked steps of genes transduction and chemically defined medium induction.
Abstract:
The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.
Abstract:
Un oligonucleótido antisentido en el que el oligonucleótido es el compuesto oligonucleotídico (CMP ID NO: 626_7) en el que las letras mayúsculas representan nucleósidos de LNA beta-D- oxi, las letras minúsculas representan nucleósidos de ADN, todos los C de LNA son 5-metil-citosina y todos los enlaces internucleosídicos son enlaces internucleosídicos de fosforotioato.
Abstract:
The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.