An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
Saved in:
| Title: | An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion. |
|---|---|
| Authors: | Keller CG; Novartis Institutes for Biomedical Research, Basel, Switzerland., Shin Y; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Monteys AM; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA., Renaud N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Beibel M; Novartis Institutes for Biomedical Research, Basel, Switzerland., Teider N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Peters T; Novartis Institutes for Biomedical Research, Basel, Switzerland., Faller T; Novartis Institutes for Biomedical Research, Basel, Switzerland., St-Cyr S; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA., Knehr J; Novartis Institutes for Biomedical Research, Basel, Switzerland., Roma G; Novartis Institutes for Biomedical Research, Basel, Switzerland., Reyes A; Novartis Institutes for Biomedical Research, Basel, Switzerland., Hild M; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Lukashev D; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Theil D; Novartis Institutes for Biomedical Research, Basel, Switzerland., Dales N; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Cha JH; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Borowsky B; Novartis Pharmaceuticals, East Hanover, NJ, USA., Dolmetsch R; Novartis Institutes for Biomedical Research, Cambridge, MA, USA., Davidson BL; The Raymond G Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA. davidsonbl@chop.edu.; Department of Pathology and Laboratory Medicine, The Perelman School of Medicine, The University of Pennsylvania, Philadelphia, PA, USA. davidsonbl@chop.edu., Sivasankaran R; Novartis Institutes for Biomedical Research, Cambridge, MA, USA. rajeev.sivasankaran@novartis.com. |
| Source: | Nature communications [Nat Commun] 2022 Mar 03; Vol. 13 (1), pp. 1150. Date of Electronic Publication: 2022 Mar 03. |
| Publication Type: | Journal Article; Clinical Trial, Phase II; Clinical Trial, Phase I |
| Journal Info: | Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
|
Full text is not displayed to guests.
Login for full access.
|
|
| ISSN: | 2041-1723 |
|---|---|
| DOI: | 10.1038/s41467-022-28653-6 |
