Hoffmann-la Roche Inc.

Roche Innovation Center Copenhagen A/s

F. Hoffmann-la Roche Ag

Christoph Patsch

Marius Hoener

Lykke Pedersen

Maj Hedtjärn

Søren Vestergaard Rasmussen

Veronica Costa

Mads Aaboe Jensen

Ravi Jagasia

Roberto Iacone

Martin Graf

Jacques Bailly

Eva Christina Thoma

Osele Ciampi

Klaus Christensen

Soren Vestergaard Rasmussen

Maj Hedtjarn

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 SNORD1091B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

Oligonucleotides for inducing paternal UBE3A expression

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.

This application relates to a method for differentiating pluripotent stem cells (PSCs) into multi-competent renal precursor cells expressing Six2. These renal precursor cells are able to differentiate into fully functional and fully differentiated podocytes. Moreover this application relates to a method for differentiating human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) into defined renal precursor cells expressing Six2 and podocytes based on linked steps of chemically defined medium inductions.

Method for differentiation of pluripotent stem cells into multi-competent renal precursors

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Basel, Switzerland

Christoph Patsch